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1. Treatment of Multisystem Inflammatory Syndrome in Children

Author: McArdle A. J., Vito O., Patel H., Seaby E. G., Shah P., Wilson C., Broderick C., Nijman R., Tremoulet A. H., Munblit D., Ulloa-Gutierrez R., Carter M. J., De T., Hoggart C., Whittaker E., Herberg J. A., Kaforou M., Cunnington A. J., Levin M., Vazquez J. A., Carmona R., Perez L., Rubinos M., Veliz N., Yori S., Haerynck F., Hoste L., Leal I. A., Da Silva A. R. A., Silva A. E. A., Barchik A., Barreiro S. T. A., Cochrane N., Teixeira C. H., Arauj J. M., Ossa R. A. P. -D. L., Vieira C. S., Dimitrova A., Ganeva M., Stefanov S., Telcharova-Mihaylovska A., Biggs C. M., Scuccimarri R., Withington D., Raul B. B., Ampuero C., Aravena J., Casanova D., Cruces P., Diaz F., Garcia-Salum T., Godoy L., Medina R. A., Galaz G. V., Avila-Aguero M. L., Brenes-Chacon H., Ivankovich-Escoto G., Yock-Corrales A., Badib A., Badreldin K., Elkhashab Y., Heshmat H., Heinonen S., Angoulvant F., Belot A., Ouldali N., Beske F., Heep A., Masjosthusmann K., Reiter K., Heuvel I. V. D., Both U. V., Agrafiotou A., Antachopoulos C., Eleftheriou I., Farmaki E., Fotis L., Kafetzis D., Lampidi S., Liakopoulou T., Maritsi D., Michailidou E., Milioudi M., Mparmpounaki I., Papadimitriou E., Papaevangelou V., Roilides E., Tsiatsiou O., Tsolas G., Tsolia M., Vantsi P., Pineda L. Y. B., Aguilar K. L. B., Quintero E. M. C., Ip P., Kwan M. Y. W., Kwok J., Lau Y. L., To K., Wong J. S. C., David M., Farkas D., Kalcakosz S., Szekeres K., Zsigmond B., Aslam N., Andreozzi L., Bianco F., Bucciarelli V., Buonsenso D., Cimaz R., D'Argenio P., Dellepiane R. M., Fabi M., Mastrolia M. V., Mauro A., Mazza A., Romani L., Simonini G., Tipo V., Valentini P., Verdoni L., Reel B., Pace D., Torpiano P., Flores M. F., Dominguez M. G., Vargas A. L. G., Hernandez L. L., Figueroa R. P. M., Gaxiola G. P., Valadez J., Klevberg S., Knudsen P. K., Maseide P. H., Carrera J. M., Castano E. G., Timana C. A. D., Leon T. D., Estripeaut D., Levy J., Norero X., Record J., Rojas-Bonilla M., Iramain R., Hernandez R., Huaman G., Munaico M., Peralta C., Seminario D., Yarleque E. H. Z., Gadzinska J., Mandziuk J., Okarska-Napierala M., Alacheva Z. A., Alexeeva E., Ananin P. V., Antsupova M., Bakradze M. D., Bobkova P., Borzakova S., Chashchina I. L., Fisenko A. P., Gautier M. S., Glazyrina A., Kondrikova E., Korobyants E., Korsunskiy A. A., Kovygina K., Krasnaya E., Kurbanova S., Kurdup M. K., Mamutova A. V., Mazankova L., Mitushin I. L., Nargizyan A., Orlova Y. O., Osmanov I. M., Polyakova A. S., Romanova O., Samitova E., Sologub A., Spiridonova E., Tepaev R. F., Tkacheva A. A., Yusupova V., Zholobova E., Grasa C. D., Segura N. L., Martinon-Torres F., Melendo S., Echevarria A. M., Guzman J. M. M., Argueta J. R. P., Rivero-Calle I., Riviere J., Rodriguez-Gonzalez M., Rojo P., Manubens J. S., Soler-Palacin P., Soriano-Arandes A., Tagarro A., Villaverde S., Altman M., Brodin P., Horne A., Palmblad K., Brotschi B., Sauteur P. M., Schmid J. P., Prader S., Relly C., Schlapbach L. J., Seiler M., Truck J., Wutz D., Ketharanathan N., Vermont C., Ozkan E. A., Erdeniz E. H., Borisova G., Boychenko L., Diudenko N., Kasiyan O., Katerynych K., Melnyk K., Miagka N., Teslenko M., Trykosh M., Volokha A., Akomolafe T., Al-Abadi E., Alders N., Avram P., Bamford A., Bank M., Roy R. B., Beattie T., Boleti O., Broad J., Carrol E. D., Chandran A., Cooper H., Davies P., Emonts M., Evans C., Fidler K., Foster C., Gong C., Gongrun B., Gonzalez C., Grandjean L., Grant K., Hacohen Y., Hall J., Hassell J., Hesketh C., Hewlett J., Hnieno A., Holt-Davis H., Hossain A., Hudson L. D., Johnson M., Johnson S., Jyothish D., Kampmann B., Kavirayani A., Kelly D., Kucera F., Langer D., Lillie J., Longbottom K., Lyall H., MacKdermott N., Maltby S., McLelland T., McMahon A. -M., Miller D., Morrison Z., Mosha K., Muller J., Myttaraki E., Nadel S., Osaghae D., Osman F., Ostrzewska A., Panthula M., Papachatzi E., Papadopoulou C., Penner J., Polandi S., Prendergast A. J., Ramnarayan P., Rhys-Evans S., Riordan A., Rodrigues C. M. C., Romaine S., Seddon J., Shingadia D., Srivastava A., Struik S., Taylor A., Tran S., Tudor-Williams G., Van Der Velden F., Ventilacion L., Wellman P. A., Yanney M. P., Yeung S., Badheka A., Badran S., Bailey D. M., Burch A. K., Burns J. C., Cichon C., Cirks B., Dallman M. D., Delany D. R., Fairchok M., Friedman S., Geracht J., Langs-Barlow A., Mann K., Padhye A., Quade A., Ramirez K. A., Rockett J., Sayed I. A., Shahin A. A., Umaru S., Widener R., Angela M. H., Kandawasvika G., McArdle A.J., Vito O., Patel H., Seaby E.G., Shah P., Wilson C., Broderick C., Nijman R., Tremoulet A.H., Munblit D., Ulloa-Gutierrez R., Carter M.J., De T., Hoggart C., Whittaker E., Herberg J.A., Kaforou M., Cunnington A.J., Levin M., Vazquez J.A., Carmona R., Perez L., Rubinos M., Veliz N., Yori S., Haerynck F., Hoste L., Leal I.A., Da Silva A.R.A., Silva A.E.A., Barchik A., Barreiro S.T.A., Cochrane N., Teixeira C.H., Arauj J.M., Ossa R.A.P.-D.L., Vieira C.S., Dimitrova A., Ganeva M., Stefanov S., Telcharova-Mihaylovska A., Biggs C.M., Scuccimarri R., Withington D., Raul B.B., Ampuero C., Aravena J., Casanova D., Cruces P., Diaz F., Garcia-Salum T., Godoy L., Medina R.A., Galaz G.V., Avila-Aguero M.L., Brenes-Chacon H., Ivankovich-Escoto G., Yock-Corrales A., Badib A., Badreldin K., Elkhashab Y., Heshmat H., Heinonen S., Angoulvant F., Belot A., Ouldali N., Beske F., Heep A., Masjosthusmann K., Reiter K., Heuvel I.V.D., Both U.V., Agrafiotou A., Antachopoulos C., Eleftheriou I., Farmaki E., Fotis L., Kafetzis D., Lampidi S., Liakopoulou T., Maritsi D., Michailidou E., Milioudi M., Mparmpounaki I., Papadimitriou E., Papaevangelou V., Roilides E., Tsiatsiou O., Tsolas G., Tsolia M., Vantsi P., Pineda L.Y.B., Aguilar K.L.B., Quintero E.M.C., Ip P., Kwan M.Y.W., Kwok J., Lau Y.L., To K., Wong J.S.C., David M., Farkas D., Kalcakosz S., Szekeres K., Zsigmond B., Aslam N., Andreozzi L., Bianco F., Bucciarelli V., Buonsenso D., Cimaz R., D'Argenio P., Dellepiane R.M., Fabi M., Mastrolia M.V., Mauro A., Mazza A., Romani L., Simonini G., Tipo V., Valentini P., Verdoni L., Reel B., Pace D., Torpiano P., Flores M.F., Dominguez M.G., Vargas A.L.G., Hernandez L.L., Figueroa R.P.M., Gaxiola G.P., Valadez J., Klevberg S., Knudsen P.K., Maseide P.H., Carrera J.M., Castano E.G., Timana C.A.D., Leon T.D., Estripeaut D., Levy J., Norero X., Record J., Rojas-Bonilla M., Iramain R., Hernandez R., Huaman G., Munaico M., Peralta C., Seminario D., Yarleque E.H.Z., Gadzinska J., Mandziuk J., Okarska-Napierala M., Alacheva Z.A., Alexeeva E., Ananin P.V., Antsupova M., Bakradze M.D., Bobkova P., Borzakova S., Chashchina I.L., Fisenko A.P., Gautier M.S., Glazyrina A., Kondrikova E., Korobyants E., Korsunskiy A.A., Kovygina K., Krasnaya E., Kurbanova S., Kurdup M.K., Mamutova A.V., Mazankova L., Mitushin I.L., Nargizyan A., Orlova Y.O., Osmanov I.M., Polyakova A.S., Romanova O., Samitova E., Sologub A., Spiridonova E., Tepaev R.F., Tkacheva A.A., Yusupova V., Zholobova E., Grasa C.D., Segura N.L., Martinon-Torres F., Melendo S., Echevarria A.M., Guzman J.M.M., Argueta J.R.P., Rivero-Calle I., Riviere J., Rodriguez-Gonzalez M., Rojo P., Manubens J.S., Soler-Palacin P., Soriano-Arandes A., Tagarro A., Villaverde S., Altman M., Brodin P., Horne A., Palmblad K., Brotschi B., Sauteur P.M., Schmid J.P., Prader S., Relly C., Schlapbach L.J., Seiler M., Truck J., Wutz D., Ketharanathan N., Vermont C., Ozkan E.A., Erdeniz E.H., Borisova G., Boychenko L., Diudenko N., Kasiyan O., Katerynych K., Melnyk K., Miagka N., Teslenko M., Trykosh M., Volokha A., Akomolafe T., Al-Abadi E., Alders N., Avram P., Bamford A., Bank M., Roy R.B., Beattie T., Boleti O., Broad J., Carrol E.D., Chandran A., Cooper H., Davies P., Emonts M., Evans C., Fidler K., Foster C., Gong C., Gongrun B., Gonzalez C., Grandjean L., Grant K., Hacohen Y., Hall J., Hassell J., Hesketh C., Hewlett J., Hnieno A., Holt-Davis H., Hossain A., Hudson L.D., Johnson M., Johnson S., Jyothish D., Kampmann B., Kavirayani A., Kelly D., Kucera F., Langer D., Lillie J., Longbottom K., Lyall H., MacKdermott N., Maltby S., McLelland T., McMahon A.-M., Miller D., Morrison Z., Mosha K., Muller J., Myttaraki E., Nadel S., Osaghae D., Osman F., Ostrzewska A., Panthula M., Papachatzi E., Papadopoulou C., Penner J., Polandi S., Prendergast A.J., Ramnarayan P., Rhys-Evans S., Riordan A., Rodrigues C.M.C., Romaine S., Seddon J., Shingadia D., Srivastava A., Struik S., Taylor A., Tran S., Tudor-Williams G., Van Der Velden F., Ventilacion L., Wellman P.A., Yanney M.P., Yeung S., Badheka A., Badran S., Bailey D.M., Burch A.K., Burns J.C., Cichon C., Cirks B., Dallman M.D., Delany D.R., Fairchok M., Friedman S., Geracht J., Langs-Barlow A., Mann K., Padhye A., Quade A., Ramirez K.A., Rockett J., Sayed I.A., Shahin A.A., Umaru S., Widener R., Angela M.H., Kandawasvika G., Pediatric Surgery, Pediatrics, University of Zurich, National Institute of Health and Medical Research, Wellcome Trust, Medical Research Foundation, Shah, Priyen [0000-0001-9164-8862], Ulloa-Gutierrez, Rolando [0000-0002-9157-9227], Herberg, Jethro A [0000-0001-6941-6491], Cunnington, Aubrey J [0000-0002-1305-3529], Levin, Michael [0000-0003-2767-6919], and Apollo - University of Cambridge Repository
Subjects: Inotrope, Male, medicine.medical_treatment, 2700 General Medicine, 030204 cardiovascular system & hematology, Antibodies, Viral, Medical and Health Sciences, Cohort Studies, 0302 clinical medicine, Glucocorticoid, hemic and lymphatic diseases, Medicine and Health Sciences, 030212 general & internal medicine, Viral, Child, 11 Medical and Health Sciences, OUTCOMES, Respiration, Immunoglobulins, Intravenous, General Medicine, Systemic Inflammatory Response Syndrome, 3. Good health, Hospitalization, Treatment Outcome, Child, Preschool, Combination, Artificial, Regression Analysis, Drug Therapy, Combination, Female, Original Article, Intravenous, Life Sciences & Biomedicine, Cohort study, Human, medicine.medical_specialty, BATS Consortium, Adolescent, Immunoglobulins, 610 Medicine & health, Regression Analysi, Antibodies, Immunomodulation, 03 medical and health sciences, Medicine, General & Internal, Pharmacotherapy, Drug Therapy, Clinical Research, Internal medicine, General & Internal Medicine, medicine, MANAGEMENT, Confidence Intervals, Humans, Preschool, Propensity Score, Glucocorticoids, Mechanical ventilation, Science & Technology, business.industry, SARS-CoV-2, Inflammatory and immune system, COVID-19, Odds ratio, medicine.disease, Respiration, Artificial, Confidence interval, KAWASAKI-LIKE DISEASE, COVID-19 Drug Treatment, Systemic inflammatory response syndrome, 10036 Medical Clinic, Immunoglobulins, Intravenou, Propensity score matching, Cohort Studie, business, ACUTE RESPIRATORY SYNDROME, Confidence Interval, TOXIC-SHOCK-SYNDROME
Abstract: BackgroundEvidence is urgently needed to support treatment decisions for children with multisystem inflammatory syndrome (MIS-C) associated with severe acute respiratory syndrome coronavirus 2.MethodsWe performed an international observational cohort study of clinical and outcome data regarding suspected MIS-C that had been uploaded by physicians onto a Web-based database. We used inverse-probability weighting and generalized linear models to evaluate intravenous immune globulin (IVIG) as a reference, as compared with IVIG plus glucocorticoids and glucocorticoids alone. There were two primary outcomes: the first was a composite of inotropic support or mechanical ventilation by day 2 or later or death; the second was a reduction in disease severity on an ordinal scale by day 2. Secondary outcomes included treatment escalation and the time until a reduction in organ failure and inflammation.ResultsData were available regarding the course of treatment for 614 children from 32 countries from June 2020 through February 2021; 490 met the World Health Organization criteria for MIS-C. Of the 614 children with suspected MIS-C, 246 received primary treatment with IVIG alone, 208 with IVIG plus glucocorticoids, and 99 with glucocorticoids alone; 22 children received other treatment combinations, including biologic agents, and 39 received no immunomodulatory therapy. Receipt of inotropic or ventilatory support or death occurred in 56 patients who received IVIG plus glucocorticoids (adjusted odds ratio for the comparison with IVIG alone, 0.77; 95% confidence interval [CI], 0.33 to 1.82) and in 17 patients who received glucocorticoids alone (adjusted odds ratio, 0.54; 95% CI, 0.22 to 1.33). The adjusted odds ratios for a reduction in disease severity were similar in the two groups, as compared with IVIG alone (0.90 for IVIG plus glucocorticoids and 0.93 for glucocorticoids alone). The time until a reduction in disease severity was similar in the three groups.ConclusionsWe found no evidence that recovery from MIS-C differed after primary treatment with IVIG alone, IVIG plus glucocorticoids, or glucocorticoids alone, although significant differences may emerge as more data accrue. (Funded by the European Union's Horizon 2020 Program and others; BATS ISRCTN number, ISRCTN69546370.).
Published: 2021

2. Treatment of Multisystem Inflammatory Syndrome in Children

Author: McArdle, A.J. Vito, O. Patel, H. Seaby, E.G. Shah, P. Wilson, C. Broderick, C. Nijman, R. Tremoulet, A.H. Munblit, D. Ulloa-Gutierrez, R. Carter, M.J. De, T. Hoggart, C. Whittaker, E. Herberg, J.A. Kaforou, M. Cunnington, A.J. Levin, M. Vazquez, J.A. Carmona, R. Perez, L. Rubinos, M. Veliz, N. Yori, S. Haerynck, F. Hoste, L. Leal, I.A. Da Silva, A.R.A. Silva, A.E.A. Barchik, A. Barreiro, S.T.A. Cochrane, N. Teixeira, C.H. Arauj, J.M. Ossa, R.A.P.-D.L. Vieira, C.S. Dimitrova, A. Ganeva, M. Stefanov, S. Telcharova-Mihaylovska, A. Biggs, C.M. Scuccimarri, R. Withington, D. Raul, B.B. Ampuero, C. Aravena, J. Casanova, D. Cruces, P. Diaz, F. Garcia-Salum, T. Godoy, L. Medina, R.A. Galaz, G.V. Avila-Aguero, M.L. Brenes-Chacon, H. Ivankovich-Escoto, G. Yock-Corrales, A. Badib, A. Badreldin, K. Elkhashab, Y. Heshmat, H. Heinonen, S. Angoulvant, F. Belot, A. Ouldali, N. Beske, F. Heep, A. Masjosthusmann, K. Reiter, K. Heuvel, I.V.D. Both, U.V. Agrafiotou, A. Antachopoulos, C. Eleftheriou, I. Farmaki, E. Fotis, L. Kafetzis, D. Lampidi, S. Liakopoulou, T. Maritsi, D. Michailidou, E. Milioudi, M. Mparmpounaki, I. Papadimitriou, E. Papaevangelou, V. Roilides, E. Tsiatsiou, O. Tsolas, G. Tsolia, M. Vantsi, P. Pineda, L.Y.B. Aguilar, K.L.B. Quintero, E.M.C. Ip, P. Kwan, M.Y.W. Kwok, J. Lau, Y.L. To, K. Wong, J.S.C. David, M. Farkas, D. Kalcakosz, S. Szekeres, K. Zsigmond, B. Aslam, N. Andreozzi, L. Bianco, F. Bucciarelli, V. Buonsenso, D. Cimaz, R. D'Argenio, P. Dellepiane, R.M. Fabi, M. Mastrolia, M.V. Mauro, A. Mazza, A. Romani, L. Simonini, G. Tipo, V. Valentini, P. Verdoni, L. Reel, B. Pace, D. Torpiano, P. Flores, M.F. Domínguez, M.G. Vargas, A.L.G. Hernandez, L.L. Figueroa, R.P.M. Gaxiola, G.P. Valadez, J. Klevberg, S. Knudsen, P.K. Maseide, P.H. Carrera, J.M. Castano, E.G. Timana, C.A.D. Leon, T.D. Estripeaut, D. Levy, J. Norero, X. Record, J. Rojas-Bonilla, M. Iramain, R. Hernandez, R. Huaman, G. Munaico, M. Peralta, C. Seminario, D. Yarleque, E.H.Z. Gadzinska, J. Mandziuk, J. Okarska-Napierała, M. Alacheva, Z.A. Alexeeva, E. Ananin, P.V. Antsupova, M. Bakradze, M.D. Bobkova, P. Borzakova, S. Chashchina, I.L. Fisenko, A.P. Gautier, M.S. Glazyrina, A. Kondrikova, E. Korobyants, E. Korsunskiy, A.A. Kovygina, K. Krasnaya, E. Kurbanova, S. Kurdup, M.K. Mamutova, A.V. Mazankova, L. Mitushin, I.L. Nargizyan, A. Orlova, Y.O. Osmanov, I.M. Polyakova, A.S. Romanova, O. Samitova, E. Sologub, A. Spiridonova, E. Tepaev, R.F. Tkacheva, A.A. Yusupova, V. Zholobova, E. Grasa, C.D. Segura, N.L. Martinon-Torres, F. Melendo, S. Echevarria, A.M. Guzman, J.M.M. Argueta, J.R.P. Rivero-Calle, I. Riviere, J. Rodriguez-Gonzalez, M. Rojo, P. Manubens, J.S. Soler-Palacin, P. Soriano-Arandes, A. Tagarro, A. Villaverde, S. Altman, M. Brodin, P. Horne, A. Palmblad, K. Brotschi, B. Sauteur, P.M. Schmid, J.P. Prader, S. Relly, C. Schlapbach, L.J. Seiler, M. Truck, J. Wutz, D. Ketharanathan, N. Vermont, C. Ozkan, E.A. Erdeniz, E.H. Borisova, G. Boychenko, L. Diudenko, N. Kasiyan, O. Katerynych, K. Melnyk, K. Miagka, N. Teslenko, M. Trykosh, M. Volokha, A. Akomolafe, T. Al-Abadi, E. Alders, N. Avram, P. Bamford, A. Bank, M. Roy, R.B. Beattie, T. Boleti, O. Broad, J. Carrol, E.D. Chandran, A. Cooper, H. Davies, P. Emonts, M. Evans, C. Fidler, K. Foster, C. Gong, C. Gongrun, B. Gonzalez, C. Grandjean, L. Grant, K. Hacohen, Y. Hall, J. Hassell, J. Hesketh, C. Hewlett, J. Hnieno, A. Holt-Davis, H. Hossain, A. Hudson, L.D. Johnson, M. Johnson, S. Jyothish, D. Kampmann, B. Kavirayani, A. Kelly, D. Kucera, F. Langer, D. Lillie, J. Longbottom, K. Lyall, H. MacKdermott, N. Maltby, S. McLelland, T. McMahon, A.-M. Miller, D. Morrison, Z. Mosha, K. Muller, J. Myttaraki, E. Nadel, S. Osaghae, D. Osman, F. Ostrzewska, A. Panthula, M. Papachatzi, E. Papadopoulou, C. Penner, J. Polandi, S. Prendergast, A.J. Ramnarayan, P. Rhys-Evans, S. Riordan, A. Rodrigues, C.M.C. Romaine, S. Seddon, J. Shingadia, D. Srivastava, A. Struik, S. Taylor, A. Taylor, A. Taylor, A. Tran, S. Tudor-Williams, G. Van Der Velden, F. Ventilacion, L. Wellman, P.A. Yanney, M.P. Yeung, S. Badheka, A. Badran, S. Bailey, D.M. Burch, A.K. Burns, J.C. Cichon, C. Cirks, B. Dallman, M.D. Delany, D.R. Fairchok, M. Friedman, S. Geracht, J. Langs-Barlow, A. Mann, K. Padhye, A. Quade, A. Ramirez, K.A. Rockett, J. Sayed, I.A. Shahin, A.A. Umaru, S. Widener, R. Angela, M.H. Kandawasvika, G. BATS Consortium
Subjects: hemic and lymphatic diseases
Abstract: BACKGROUND Evidence is urgently needed to support treatment decisions for children with multisystem inflammatory syndrome (MIS-C) associated with severe acute respiratory syndrome coronavirus 2. METHODS We performed an international observational cohort study of clinical and outcome data regarding suspected MIS-C that had been uploaded by physicians onto a Web-based database. We used inverse-probability weighting and generalized linear models to evaluate intravenous immune globulin (IVIG) as a reference, as compared with IVIG plus glucocorticoids and glucocorticoids alone. There were two primary outcomes: the first was a composite of inotropic support or mechanical ventilation by day 2 or later or death; the second was a reduction in disease severity on an ordinal scale by day 2. Secondary outcomes included treatment escalation and the time until a reduction in organ failure and inflammation. RESULTS Data were available regarding the course of treatment for 614 children from 32 countries from June 2020 through February 2021; 490 met the World Health Organization criteria for MIS-C. Of the 614 children with suspected MIS-C, 246 received primary treatment with IVIG alone, 208 with IVIG plus glucocorticoids, and 99 with glucocorticoids alone; 22 children received other treatment combinations, including biologic agents, and 39 received no immunomodulatory therapy. Receipt of inotropic or ventilatory support or death occurred in 56 patients who received IVIG plus glucocorticoids (adjusted odds ratio for the comparison with IVIG alone, 0.77; 95% confidence interval [CI], 0.33 to 1.82) and in 17 patients who received glucocorticoids alone (adjusted odds ratio, 0.54; 95% CI, 0.22 to 1.33). The adjusted odds ratios for a reduction in disease severity were similar in the two groups, as compared with IVIG alone (0.90 for IVIG plus glucocorticoids and 0.93 for glucocorticoids alone). The time until a reduction in disease severity was similar in the three groups. CONCLUSIONS We found no evidence that recovery from MIS-C differed after primary treatment with IVIG alone, IVIG plus glucocorticoids, or glucocorticoids alone, although significant differences may emerge as more data accrue. Copyright © 2021 Massachusetts Medical Society.
Published: 2021

3. Use of linezolid in neonatal and pediatric inpatient facilities—results of a retrospective multicenter survey

Author: Simon, A., Müllenborn, E., Prelog, M., Schenk, W., Holzapfel, J., Ebinger, F., Klabunde-Cherwon, A., Faber, J., Groll, A. H., Masjosthusmann, K., Dohna-Schwake, C., Beutel, K., Dirkwinkel, E., Lehrnbecher, T., Ammann, R. A., and Müller, A.
Published: 2012
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4. Risikomanagement durch CIRS-Analyse: Fehleranalyse durch ein freiwilliges, anonymes „critical incident reporting system“ auf einer neonatologischen und interdisziplinären pädiatrischen Intensivstation

Author: Berghäuser, M.A., Masjosthusmann, K., and Rellensmann, G.
Published: 2010
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5. Delay in B-lymphocyte recovery and function following rituximab for EBV-associated lymphoproliferative disease early post-allogeneic hematopoietic SCT

Author: Masjosthusmann, K, Ehlert, K, Eing, B R, Roth, J, Koehler, G, Juergens, H, Fruehwald, M, and Groll, A H
Published: 2009
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6. The German national registry for primary immunodeficiencies (PID)

Author: Gathmann, B., Goldacker, S., Klima, M., Belohradsky, B. H., Notheis, G., Ehl, S., Ritterbusch, H., Baumann, U., Meyer-Bahlburg, A., Witte, T., Schmidt, R., Borte, M., Borte, S., Linde, R., Schubert, R., Bienemann, K., Laws, H.-J., Dueckers, G., Roesler, J., Rothoeft, T., Krüger, R., Scharbatke, E. C., Masjosthusmann, K., Wasmuth, J.-C., Moser, O., Kaiser, P., Gro-Wieltsch, U., Classen, C. F., Horneff, G., Reiser, V., Binder, N., El-Helou, S. M., Klein, C., Grimbacher, B., and Kindle, G.
Published: 2013
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7. Fatal human metapneumovirus infection following allogeneic hematopoietic stem cell transplantation

Author: Dokos, C., Masjosthusmann, K., Rellensmann, G., Werner, C., Schuler-Lüttmann, S., Müller, K.-M., Schiborr, M., Ehlert, K., and Groll, A. H.
Published: 2013
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8. A child with soft-tissue infection and lymphadenitis

Author: Froböse, N.J., primary, Masjosthusmann, K., additional, Huss, S., additional, Correa-Martinez, C.L., additional, Mellmann, A., additional, Schuler, F., additional, Kahl, B.C., additional, Wittkowski, H., additional, and Schaumburg, F., additional
Published: 2020
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9. Delay in B-lymphocyte recovery and function followingrituximab for EBV-associated lymphoproliferative disease early post-allogeneic hematopoietic SCT

Author: Masjosthusmann, K, Ehlert, K, Eing, B R, Roth, J, Koehler, G, Juergens, H, Fruehwald, M, and Groll, A H
Published: 2009

10. The German PID-net registry

Author: El-Helou, S. M., Biegner, A. K., Bode, S., Ehl, S., Heeg, M., Ritterbusch, H., Rusch, S., Schmitt, R., Warnatz, K., Baerlecken, N. T., Baumann, U., Beider, R., Ernst, D., Gerschmann, S., Klemann, C., Mielke, G., Schmidt, R. E., Schuermann, G., Viemann, D., Albert, M., Eichinger, A., Eisl, E., Hauck, F., Klein, C., Sollinger, F., von Bernuth, H., Hanitsch, L., Krueger, R., Scheibenbogen, C., Avila, A., Borte, M., Borte, S., Fasshauer, M., Hauenherm, A., Kellner, N., Mueller, H., Uelzen, A., Bader, P., Bakhtiar, S., Hess, U., Lee, J. Y., Schubert, R., Voss, S., Zielen, S., Bienemann, K., Lankisch, P., Laws, H. J., Neubert, J., Dueckers, G., Lamers, B., Langemeyer, V., Niehues, T., Hoenig, M., Schulz, A., Schwarz, K., Steinmann, S., Graf, D., Haase, G., Liese, J. G., Morbach, H., Schwaneck, E., Tony, H. P., Foell, D., Hellige, A., Masjosthusmann, K., Mohr, M., Wittkowski, H., Fischer, J. C., Hedrich, C. M., Roesen-Wolff, A., Roesler, J., Behrends, U., Rieber, N., Schauer, U., Boesecke, C., Dilloo, D., Engelhardt, A., Huelsmann, B., Rockstroh, J., Scholtes, C., Schoenberger, S., Wasmuth, J. C., Handgretinger, R., Henes, J., Holzer, U., Kanz, L., Ankermann, T., von Bismarck, P., Schreiber, S., Zeuner, R., Huppertz, H. I., Kaiser-Labusch, P., Greil, J., Jakoby, D., Kulozik, A. E., Graf, N., Heine, S., Garwer, B., Kobbe, R., Lehmberg, K., Mueller, I., Herrmann, F., Horneff, G., Klein, A., Peitz, J., Schmidt, N., Apel, K., Bielack, S., Gross-Wieltsch, U., Deutz, P., Lassay, L., Kamitz, D., Stienen, A., Tenbrock, K., Wagner, N., Classen, C. F., Weiss, M., Bernbeck, B., Brummel, B., Lara-Villacanas, E., Muenstermann, E., Schneider, D., Tietsch, N., Westkemper, M., Naumann-Bartsch, N., Metzler, M., Sobik, B., Kuehnle, I., Kullmann, S., Kramm, C., Girschick, H., Elbe, S., Specker, C., Vinnemeier-Laubenthal, L., Haenicke, H., Schulz, C., Schweigerer, L., Mueller, T. G., Kindle, G., Grimbacher, B., El-Helou, S. M., Biegner, A. K., Bode, S., Ehl, S., Heeg, M., Ritterbusch, H., Rusch, S., Schmitt, R., Warnatz, K., Baerlecken, N. T., Baumann, U., Beider, R., Ernst, D., Gerschmann, S., Klemann, C., Mielke, G., Schmidt, R. E., Schuermann, G., Viemann, D., Albert, M., Eichinger, A., Eisl, E., Hauck, F., Klein, C., Sollinger, F., von Bernuth, H., Hanitsch, L., Krueger, R., Scheibenbogen, C., Avila, A., Borte, M., Borte, S., Fasshauer, M., Hauenherm, A., Kellner, N., Mueller, H., Uelzen, A., Bader, P., Bakhtiar, S., Hess, U., Lee, J. Y., Schubert, R., Voss, S., Zielen, S., Bienemann, K., Lankisch, P., Laws, H. J., Neubert, J., Dueckers, G., Lamers, B., Langemeyer, V., Niehues, T., Hoenig, M., Schulz, A., Schwarz, K., Steinmann, S., Graf, D., Haase, G., Liese, J. G., Morbach, H., Schwaneck, E., Tony, H. P., Foell, D., Hellige, A., Masjosthusmann, K., Mohr, M., Wittkowski, H., Fischer, J. C., Hedrich, C. M., Roesen-Wolff, A., Roesler, J., Behrends, U., Rieber, N., Schauer, U., Boesecke, C., Dilloo, D., Engelhardt, A., Huelsmann, B., Rockstroh, J., Scholtes, C., Schoenberger, S., Wasmuth, J. C., Handgretinger, R., Henes, J., Holzer, U., Kanz, L., Ankermann, T., von Bismarck, P., Schreiber, S., Zeuner, R., Huppertz, H. I., Kaiser-Labusch, P., Greil, J., Jakoby, D., Kulozik, A. E., Graf, N., Heine, S., Garwer, B., Kobbe, R., Lehmberg, K., Mueller, I., Herrmann, F., Horneff, G., Klein, A., Peitz, J., Schmidt, N., Apel, K., Bielack, S., Gross-Wieltsch, U., Deutz, P., Lassay, L., Kamitz, D., Stienen, A., Tenbrock, K., Wagner, N., Classen, C. F., Weiss, M., Bernbeck, B., Brummel, B., Lara-Villacanas, E., Muenstermann, E., Schneider, D., Tietsch, N., Westkemper, M., Naumann-Bartsch, N., Metzler, M., Sobik, B., Kuehnle, I., Kullmann, S., Kramm, C., Girschick, H., Elbe, S., Specker, C., Vinnemeier-Laubenthal, L., Haenicke, H., Schulz, C., Schweigerer, L., Mueller, T. G., Kindle, G., and Grimbacher, B.
Published: 2017

11. Fatal Human Metapneumovirus Pneumonitis post Hematopoietic Stem Cell Transplantation

Author: Dokos, C., Masjosthusmann, K., Rellensmann, G., Werner, C., Schuler-Lüttmann, S., Müller, K.-M., Holzinger, D., and Groll, A. H.
Subjects: ddc: 610, viruses, virus diseases, 610 Medical sciences, Medicine, respiratory tract diseases
Abstract: Objectives: Human Metapneumovirus (HMPV) is a common cause of respiratory viral infections in the community. Little is known about its relevance in immunocompromised children. Methods: Case report of a fatal HMPV infection in patient with graft-versus-host disease (GVHD) following hematopoietic stem[for full text, please go to the a.m. URL], 21. Jahrestagung der Deutschen Gesellschaft für Pädiatrische Infektiologie (DGPI)
Published: 2013

12. Management höhergradiger Mehrlinge als interdisziplinäre Herausforderung am Beispiel einer Fünflingsgravidität

Author: Oelmeier, K, primary, Hammer, K, additional, Möllers, M, additional, Falkenberg, M, additional, Braun, J, additional, Rieger-Fackeldey, E, additional, Masjosthusmann, K, additional, Klockenbusch, W, additional, and Schmitz, R, additional
Published: 2015
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13. ABSTRACT 374

Author: van den Heuvel, I., primary, Masjosthusmann, K., additional, Kurlemann, G., additional, Hülskamp, G., additional, and Omran, H., additional
Published: 2014
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14. Fatal Human Metapneumovirus Pneumonitis post Hematopoietic Stem Cell Transplantation

Author: Dokos, C, Masjosthusmann, K, Rellensmann, G, Werner, C, Schuler-Lüttmann, S, Müller, KM, Holzinger, D, Groll, AH, Dokos, C, Masjosthusmann, K, Rellensmann, G, Werner, C, Schuler-Lüttmann, S, Müller, KM, Holzinger, D, and Groll, AH
Published: 2013

15. Use of linezolid in neonatal and pediatric inpatient facilities—results of a retrospective multicenter survey

Author: Simon, A., primary, Müllenborn, E., additional, Prelog, M., additional, Schenk, W., additional, Holzapfel, J., additional, Ebinger, F., additional, Klabunde-Cherwon, A., additional, Faber, J., additional, Groll, A. H., additional, Masjosthusmann, K., additional, Dohna-Schwake, C., additional, Beutel, K., additional, Dirkwinkel, E., additional, Lehrnbecher, T., additional, Ammann, R. A., additional, and Müller, A., additional
Published: 2011
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16. Perinatale Asphyxie und hämorrhagischer Schock durch Ruptur eines kongenitalen Tumors

Author: Nowacki, M, primary, Masjosthusmann, K, additional, Kerl, K, additional, Lohse, K, additional, Leuschner, I, additional, Hörnig-Franz, I, additional, and Frühwald, M, additional
Published: 2010
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17. Erstmanifestation einer Mitochondriopathie als Ursache maligner Herzrhythmusstörungen und kardialer Dekompensation

Author: Masjosthusmann, K, primary, Sandkötter, J, additional, Kotthoff, S, additional, Scheld, HH, additional, Rellensmann, G, additional, Werner, C, additional, and Kehl, HG, additional
Published: 2010
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18. Nierenagenesie in Kombination mit einer schweren Aganglionose

Author: Sandkötter, J, primary, Masjosthusmann, K, additional, Berghäuser, MA, additional, Konrad, M, additional, Funke, K, additional, Müller, V, additional, Werner, C, additional, and Rellensmann, G, additional
Published: 2010
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19. Methoden zur Energie- und Nährstoffbilanzierung bei Kindern nach hämatopoetischer Stammzelltransplantation

Author: Bauer, J, primary, Masjosthusmann, K, additional, Berghäuser, MA, additional, Werner, C, additional, Rössig, C, additional, and Frühwald, M, additional
Published: 2009
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20. Neonatale myotone Dystrophie (CDM) als Ursache fetaler Akinesie, Lungenhypoplasie, Oligohydramnion und Arthrogryposis multiplex

Author: Berghäuser, MA, primary, Masjosthusmann, K, additional, Werner, C, additional, Rellensmann, G, additional, Steinhard, J, additional, and Bauer, J, additional
Published: 2009
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21. Das EKG als wegweisendes Diagnostikum beim Bland-White-Garland Syndrom

Author: Masjosthusmann, K, primary, Berghäuser, MA, additional, Rellensmann, G, additional, Werner, C, additional, Stege, D, additional, and Bauer, J, additional
Published: 2009
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22. Energieverluste bei Muttermilch- und Formulaernährte Frühgeborenen–Gibt es Unterschiede?

Author: Bauer, J, primary, Masjosthusmann, K, additional, Berghäuser, MA, additional, Werner, C, additional, Rellensmann, G, additional, and Hörnig-Franz, I, additional
Published: 2009
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23. Inhalationsnarkose mit Sevofluran als Alternative bei Versagen konventioneller Sedierung

Author: Masjosthusmann, K, primary, Berghäuser, MA, additional, Schmidt, C, additional, Bauer, J, additional, Werner, C, additional, and Rellensmann, G, additional
Published: 2009
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24. Delay in B-lymphocyte recovery and function following rituximab for EBV-associated lymphoproliferative disease early post-allogeneic hematopoietic SCT

Author: Masjosthusmann, K, primary, Ehlert, K, additional, Eing, B R, additional, Roth, J, additional, Koehler, G, additional, Juergens, H, additional, Fruehwald, M, additional, and Groll, A H, additional
Published: 2008
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25. Voluntary anonymous Critical Incident Reporting System in a large pediatric intensive care unit. Analysis of 189 reports concerning 231 incidents

Author: Berghäuser, MA, primary, Masjosthusmann, K, additional, and Rellensmann, G, additional
Published: 2008
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26. Ein besseres Modell zum Verständnis des Säure-Basen Haushalts

Author: Rellensmann, G, primary, Masjosthusmann, K, additional, Konrad, M, additional, Hörnig-Franz, I, additional, and Marquardt, T, additional
Published: 2008
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27. Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations

Author: Tim Niehues, Catherine Waruiru, Conleth Feighery, Uwe Schauer, Virginie Courteille, Kai Lehmberg, Ingo Müller, I. Esteves, Henner Morbach, Michael Borte, Patrick Hundsdoerfer, Klaus Schwarz, Ewelina Gowin, Alessandro Aiuti, Andreas Holbro, Federica Barzaghi, João Farela Neves, Dagmar Graf, Hannah Tamary, Veneta Milenova, Benedikt Boetticher, Eleonora Gambineri, Vera Goda, Alia Eldash, Jan-Christian Wasmuth, Fabio Candotti, Svetlana O. Sharapova, Markus Metzler, Juergen Brunner, Anna Hilfanova, Brindusa Ruxandra Capilna, Pere Soler-Palacín, Arnau Antolí, Horst von Bernuth, Vassilios Lougaris, Maria Carrabba, Bernd H. Belohradsky, Julian Thalhammer, Nathalie de Vergnes, Peter Olbrich, Peter Kopač, Leif G. Hanitsch, Alexandra Nieters, Filomeen Haerynck, Juliana Gabzdilova, Sezin Aydemir, Rabab El Hawary, Patrick F.K. Yong, Maria Giovanna Danieli, Alberto Tommasini, Sandra Steinmann, Ulrich Baumann, Figen Dogu, Elisabeth Förster-Waldl, Carolina Marasco, Donato Amodio, Lorenzo Lodi, Xavier Solanich, Caterina Cancrini, Brigita Sitkauskiene, Torsten Witte, Clementina Vanessa, Nima Rezaei, Jean-Christophe Goffard, Kirsten Wittke, Emmanouil Liatsis, Helen Baxendale, Susana L. Silva, Bodo Grimbacher, Henrike Ritterbusch, Evangelia Farmaki, Safa Meshaal, Sujal Ghosh, Larysa Kostyuchenko, David Edgar, Simone Cesaro, R Zeuner, Nerea Salmón Rodríguez, Isabella Quinti, Stephan Ehl, Pauline Brosselin, Joerg C. Henes, Pilar Llobet Agulló, Rosa Maria Dellepiane, Andrea Meinhardt, Marina Kojić, Georgios Sogkas, Stephan Borte, Catharina Schuetz, Suheyla Ocak, Karin Marschall, Lukas M. Gasteiger, Stefan Raffac, Sofia Tantou, Sadia Noorani, Matthaios Speletas, Philippe Randrianomenjanahary, Ursula Holzer, Ayca Kiykim, Johannes G. Liese, Angelo Vacca, Gisela Fecker, Ekrem Unal, Koen J. van Aerde, Alba Parra-Martínez, Kaan Boztug, Sophie Stiehler, Sybille Landwehr-Kenzel, Claudio Pignata, Jennifer Neubert, Janine Reichenbach, Shahnaz Parvin, Sarah Goddard, Andrea Schroll, Dirk Holzinger, Asghar Aghamohammadi, Hassan Abolhassani, Johannes Trück, Estela Paz-Artal, Shereen M. Reda, Anna Shcherbina, Maria Raptaki, Jaroslava Orosova, Beata Wolska-Kuśnierz, Tessa Kerre, Gerrit Ahrenstorf, Ben Zion Garty, Dirk Foell, Benjamin Becker, Ulrike F. Demel, Androniki Kapousouzi, Abraham Rutgers, Klaus Warnatz, Gemma Rocamora Blanch, Stephan Rusch, Luis M. Allende, Dalia Abd Elaziz, Safa Baris, Jorisvan Montfrans, Dominik T. Schneider, Raphael Scheible, Juana Gil-Herrera, Gerhard Kindle, Annarosa Soresina, Giovanna Fabio, Uwe Wintergerst, Emilia Faria, Maria Fasshauer, Silvia Ricci, Aisha Elmarsafy, Barbara Pietrucha, Carsten Speckmann, Nizar Mahlaoui, Ulrich Heininger, Isabelle Meyts, Matthew Buckland, Efimia Papadopoulou-Alataki, Robin Kobbe, A Herwadkar, Sebastian F. N. Bode, Ali Sobh, László Maródi, Baldassarre Martire, Chiara Azzari, Maximilian Heeg, Katja Masjosthusmann, Michael H. Albert, Matteo Chinello, Juan Luis Santos-Pérez, Aarnoud Huissoon, Tanya I. Coulter, Hendrik Schulze-Koops, Norbert Graf, Radwa Alkady, Jolanta Bernatoniene, Seraina Prader, Alenka Gagro, Joachim Roesler, Taco W. Kuijpers, Ewa Więsik-Szewczyk, Maria Elena Maccari, Conrad Ferdinand Lippert, Miriam González-Amores, Johannes Dirks, Daniel E Pleguezuelo, Christof M. Kramm, Anders Fasth, Volker Schuster, Olov Ekwall, Nikolaus Rieber, Javier Carbone, Petra Kaiser-Labusch, Diana Ernst, Lucia Augusta Baselli, Luis Ignacio Gonzalez-Granado, Maria Kanariou, Stefanie S. V. Henriet, Sigune Goldacker, Kerstin Felgentreff, Oana Joean, Fine Roosens, Fabian Hauck, Eva C. Schwaneck, Milos Jesenak, Manfred Hoenig, Lenka Kapustova, Christoph Boesecke, Alain Fischer, Sara Pereira da Silva, Julia Körholz, Ansgar Schulz, Carolynne Schwarze-Zander, Mikko Seppänen, Nermeen Galal, Nora Naumann-Bartsch, Tomaz Garcez, Peter Ciznar, Klara M. Posfay-Barbe, Zelimir Pavle Eric, Reinhold E. Schmidt, Hermann J. Girschick, Sabine Heine, Anika-Kerstin Biegner, Annick A. J. M. van de Ven, Stefan Schreiber, J. Merlijn van den Berg, Nurit Assia Batzir, Alexandra Jablonka, Kim Stol, Gregor Dückers, Antonios G.A. Kolios, Ioannis Kakkas, Christian Klemann, Marina N. Guseva, Sofia Grigoriadou, Elif Karakoc-Aydiner, Antonio Marzollo, Peter D. Arkwright, Urs C. Steiner, Sara Sebnem Kilic, Romina Dieli-Crimi, Gergely Kriván, Monika Sparber-Sauer, Marco Cazzaniga, Fulvio Porta, Paraskevi Maggina, Tomas Milota, Robbert G. M. Bredius, Martine Pergent, Klaus Tenbrock, Jana Pachlopnik Schmid, Florentia Dimitriou, Cathal Laurence Steele, Helen Bourne, Anna Bobcakova, Gerd Horneff, Judith Potjewijd, Marc Schmalzing, Tobias Ankermann, Paul Ryan, Oksana Boyarchuk, Necil Kutukculer, Carl Friedrich Classen, Zita Chovancová, Moira Thomas, Cinzia Milito, Michaela Bitzenhofer-Grüber, Faranaz Atschekzei, Eva Hlaváčková, Viviana Moschese, Julie Smet, Hans-Hartmut Peter, Carla Teixeira, Sabine M El-Helou, Suzanne de Kruijf Bazen, Helmut Wittkowski, Donate Jakoby, Marina Garcia-Prat, Esther de Vries, Richard Herriot, Sven Kracker, Alessandro Plebani, Lisa Göschl, Laura Hora Marques, Anna Sediva, Jiri Litzman, Mark M. Gompels, Renate Krüger, Şefika İlknur Kökçü Karadağ, Nadine Binder, Anna Szaflarska, Peter Jandus, Lisa Ibberson, Johann Greil, Ulf Schulze-Sturm, Mehtap Sirin, Aydan Ikinciogullari, Edyta Heropolitańska-Pliszka, Michael E. Weiss, Alla Skapenko, Lukas Wisgrill, Hana Alachkar, Uta Behrends, Silvia Sánchez-Ramón, Maria N. Hatzistilianou, Otilia Petrovicova, Darko Richter, Zoreh Nademi, Jürgen K. Rockstroh, Sohilla Lotfy, Markus G. Seidel, Timothy Ronan Leahy, Audra Blažienė, Translational Immunology Groningen (TRIGR), Paediatric Infectious Diseases / Rheumatology / Immunology, AII - Inflammatory diseases, ARD - Amsterdam Reproduction and Development, University of Zurich, Ehl, Stephan, Thalhammer, J., Kindle, G., Nieters, A., Rusch, S., Seppanen, M. R. J., Fischer, A., Grimbacher, B., Edgar, D., Buckland, M., Mahlaoui, N., Ehl, S., Boztug, K., Brunner, J., Demel, U. F., Forster-Waldl, E., Gasteiger, L. M., Goschl, L., Kojic, M., Schroll, A., Seidel, M. G., Wintergerst, U., Wisgrill, L., Sharapova, S. O., Goffard, J. -C., Kerre, T., Meyts, I., Roosens, F., Smet, J., Haerynck, F., Eric, Z. P., Milenova, V., Gagro, A., Richter, D., Chovancova, Z., Hlavackova, E., Litzman, J., Milota, T., Sediva, A., Elaziz, D. A., Alkady, R. S., El Sayed El Hawary, R., Eldash, A. S., Galal, N., Lotfy, S., Meshaal, S. S., Reda, S. M., Sobh, A., Elmarsafy, A., Brosselin, P., Courteille, V., De Vergnes, N., Kracker, S., Pergent, M., Randrianomenjanahary, P., Ahrenstorf, G., Albert, M. H., Ankermann, T., Atschekzei, F., Baumann, U., Becker, B. C., Behrends, U., Belohradsky, B. H., Biegner, A. -K., Binder, N., Bode, S. F. N., Boesecke, C., Boetticher, B., Borte, M., Borte, S., Classen, C. F., Dirks, J., Duckers, G., El-Helou, S., Ernst, D., Fasshauer, M., Fecker, G., Felgentreff, K., Foell, D., Ghosh, S., Girschick, H. J., Goldacker, S., Graf, N., Graf, D., Greil, J., Hanitsch, L. G., Hauck, F., Heeg, M., Heine, S. I., Henes, J. C., Hoenig, M., Holzer, U., Holzinger, D., Horneff, G., Hundsdoerfer, P., Jablonka, A., Jakoby, D., Joean, O., Kaiser-Labusch, P., Klemann, C., Kobbe, R., Korholz, J., Kramm, C. M., Kruger, R., Landwehr-Kenzel, S., Lehmberg, K., Liese, J. G., Lippert, C. F., Maccari, M. E., Masjosthusmann, K., Meinhardt, A., Metzler, M., Morbach, H., Muller, I., Naumann-Bartsch, N., Neubert, J., Niehues, T., Peter, H. -H., Rieber, N., Ritterbusch, H., Rockstroh, J. K., Roesler, J., Schauer, U., Scheible, R., Schmalzing, M., Schmidt, R. E., Schneider, D. T., Schreiber, S., Schuetz, C., Schulz, A., Schulze-Koops, H., Schulze-Sturm, U., Schuster, V., Schwaneck, E. C., Schwarz, K., Schwarze-Zander, C., Sirin, M., Skapenko, A., Sogkas, G., Sparber-Sauer, M., Speckmann, C., Steinmann, S., Stiehler, S., Tenbrock, K., von Bernuth, H., Warnatz, K., Wasmuth, J. -C., Weiss, M., Witte, T., Wittke, K., Wittkowski, H., Zeuner, R. A., Farmaki, E., Hatzistilianou, M. N., Kakkas, I., Kanariou, M. G., Kapousouzi, A., Liatsis, E., Maggina, P., Papadopoulou-Alataki, E., Raptaki, M., Speletas, M., Tantou, S., Goda, V., Krivan, G., Marodi, L., Abolhassani, H., Aghamohammadi, A., Rezaei, N., Feighery, C., Leahy, T. R., Ryan, P., Batzir, N. A., Garty, B. Z., Tamary, H., Aiuti, A., Amodio, D., Azzari, C., Barzaghi, F., Baselli, L. A., Cancrini, C., Carrabba, M., Cazzaniga, M., Cesaro, S., Chinello, M., Danieli, M. G., Dellepiane, R. M., Fabio, G., Gambineri, E., Lodi, L., Lougaris, V., Marasco, C., Martire, B., Marzollo, A., Milito, C., Moschese, V., Pignata, C., Plebani, A., Porta, F., Quinti, I., Ricci, S., Soresina, A., Tommasini, A., Vacca, A., Vanessa, C., Blaziene, A., Sitkauskiene, B., Gowin, E., Heropolitanska-Pliszka, E., Pietrucha, B., Szaflarska, A., Wiesik-Szewczyk, E., Wolska-Kusnierz, B., Esteves, I., Faria, E., Marques, L. H., Neves, J. F., Silva, S. L., Teixeira, C., Pereira da Silva, S., Capilna, B. R., Guseva, M. N., Shcherbina, A., Bobcakova, A., Ciznar, P., Gabzdilova, J., Jesenak, M., Kapustova, L., Orosova, J., Petrovicova, O., Raffac, S., Kopac, P., Allende, L. M., Antoli, A., Blanch, G. R., Carbone, J., Dieli-Crimi, R., Garcia-Prat, M., Gil-Herrera, J., Gonzalez-Granado, L. I., Agullo, P. L., Olbrich, P., Parra-Martinez, A., Paz-Artal, E., Pleguezuelo, D. E., Rodriguez, N. S., Sanchez-Ramon, S., Santos-Perez, J. L., Solanich, X., Soler-Palacin, P., Gonzalez-Amores, M., Ekwall, O., Fasth, A., Bitzenhofer-Gruber, M., Candotti, F., Dimitriou, F., Heininger, U., Holbro, A., Jandus, P., Kolios, A. G. A., Marschall, K., Schmid, J. P., Posfay-Barbe, K. M., Prader, S., Reichenbach, J., Steiner, U. C., Truck, J., Bredius, R. G., de Kruijf- Bazen, S., de Vries, E., Henriet, S. S. V., Kuijpers, T. W., Potjewijd, J., Rutgers, A., Stol, K., van Aerde, K. J., Van den Berg, J. M., van de Ven, A. A. J. M., Montfrans, J., Aydemir, S., Baris, S., Dogu, F., Ikinciogullari, A., Karakoc-Aydiner, E., Kilic, S. S., Kiykim, A., Kokcu Karadag, S. I., Kutukculer, N., Ocak, S., Unal, E., Boyarchuk, O., Hilfanova, A., Kostyuchenko, L. V., Alachkar, H., Arkwright, P. D., Baxendale, H. E., Bernatoniene, J., Coulter, T. I., Garcez, T., Goddard, S., Gompels, M. M., Grigoriadou, S., Herriot, R., Herwadkar, A., Huissoon, A., Ibberson, L., Nademi, Z., Noorani, S., Parvin, S., Steele, C. L., Thomas, M., Waruiru, C., Yong, P. F. K., and Bourne, H.
Subjects: 0301 basic medicine, Male, Pediatrics, syndromic, Sex Factor, Disease, registry, medicine.disease_cause, Cohort Studies, 0302 clinical medicine, Primary Immunodeficiency Disease, inborn error of immunity, Immunology and Allergy, warning signs, Age Factor, Registries, Family history, presenting symptom, Child, Primary immunodeficiency, Granuloma, autoimmune, immune dysregulation, inflammatory, Adult, Autoimmune Diseases, Female, Humans, Infections, Lymphoproliferative Disorders, Middle Aged, Primary Immunodeficiency Diseases, Sex Factors, Age Factors, 10177 Dermatology Clinic, Infections/epidemiology, 3. Good health, Settore MED/02, Warning signs, Lymphoproliferative Disorder, 2723 Immunology and Allergy, Infection, Human, medicine.medical_specialty, Immunology, 610 Medicine & health, Malignancy, primary immunodeficiency, Autoimmune Disease, 03 medical and health sciences, Immunity, Autoimmune Diseases/epidemiology, medicine, 2403 Immunology, business.industry, warning sign, Common variable immunodeficiency, Granuloma/epidemiology, Immune dysregulation, medicine.disease, Primary Immunodeficiency Diseases/epidemiology, 030104 developmental biology, Lymphoproliferative Disorders/epidemiology, Cohort Studie, business, 030215 immunology
Abstract: BACKGROUND: Inborn errors of immunity (IEI) are rare diseases, which makes diagnosis a challenge. A better description of the initial presenting manifestations should improve awareness and avoid diagnostic delay. Although increased infection susceptibility is a well-known initial IEI manifestation, less is known about the frequency of other presenting manifestations.OBJECTIVE: We sought to analyze age-related initial presenting manifestations of IEI including different IEI disease cohorts.METHODS: We analyzed data on 16,486 patients of the European Society for Immunodeficiencies Registry. Patients with autoinflammatory diseases were excluded because of the limited number registered.RESULTS: Overall, 68% of patients initially presented with infections only, 9% with immune dysregulation only, and 9% with a combination of both. Syndromic features were the presenting feature in 12%, 4% had laboratory abnormalities only, 1.5% were diagnosed because of family history only, and 0.8% presented with malignancy. Two-third of patients with IEI presented before the age of 6 years, but a quarter of patients developed initial symptoms only as adults. Immune dysregulation was most frequently recognized as an initial IEI manifestation between age 6 and 25 years, with male predominance until age 10 years, shifting to female predominance after age 40 years. Infections were most prevalent as a first manifestation in patients presenting after age 30 years.CONCLUSIONS: An exclusive focus on infection-centered warning signs would have missed around 25% of patients with IEI who initially present with other manifestations.
Published: 2021

28. Proteomic mapping identifies serum marker signatures associated with MIS-C specific hyperinflammation and cardiovascular manifestation.

Author: Reiter A, Verweyen EL, Queste E, Fuehner S, Jakob A, Masjosthusmann K, Hinze C, Wittkowski H, Foell D, Meinzer U, Melki I, and Kessel C
Subjects: Humans, Male, Female, Child, Child, Preschool, Inflammation blood, Infant, Interleukin-17 blood, TNF-Related Apoptosis-Inducing Ligand blood, Interleukin-18 blood, Adenosine Deaminase blood, Cardiovascular Diseases blood, Cardiovascular Diseases immunology, Biomarkers blood, Mucocutaneous Lymph Node Syndrome blood, Mucocutaneous Lymph Node Syndrome immunology, Proteomics methods, Systemic Inflammatory Response Syndrome blood, Systemic Inflammatory Response Syndrome immunology
Abstract: Multisystem inflammatory syndrome in children (MIS-C) shares several clinical and immunological features with Kawasaki Disease (KD) and pediatric hyperinflammation, but the immuno-phenotypic overlap among these clinical mimics is still incompletely understood. Here we analyzed serum samples from treatment-naïve patients with MIS-C (n = 31) and KD (n = 11), pediatric hyperinflammation (n = 13) and healthy controls (HC, n = 10) by proximity extension assay (PEA) to profile 184 blood biomarkers. Collectively, immunophenotypic overlap between MIS-C and hyperinflammation exceeds overlap with KD. Overexpression of IL-17A in MIS-C and KD could best separate these conditions from hyperinflammatory conditions, while those were hallmarked by overabundance of adenosin deaminase and IL-18. Depletion in serum TNF-related subfamily member 9 (TNFRSF9) and apoptosis inducing ligand (TRAIL) linked with cardiovascular manifestations and myocarditis in MIS-C. Altogether, our analysis highlights important differences in molecular marker signatures also across different MIS-C and KD cohorts and suggests several previously unidentified molecular associations in context of cardiovascular inflammation., Competing Interests: Declaration of competing interest CH has received honoraria (lecture fees) from Novartis; HW has received honoraria (lecture fees) from Novartis and Takeda, and travel support from Octapharma and CSL-Behring; DF received speaker fees/honoraria from Chugai-Roche, Novartis and SOBI as well as research support from Novartis, Pfizer and SOBI. CK has received consulting fees from Novartis and Swedish Orphan Biovitrum (SOBI) (< $10,000 each) and receives research support from Novartis (> $10,000). No other disclosures relevant to this article were reported., (Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)
Published: 2024
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29. Antimicrobial Peptides (AMPs) and the Microbiome in Preterm Infants: Consequences and Opportunities for Future Therapeutics.

Author: Marissen J, Reichert L, Härtel C, Fortmann MI, Faust K, Msanga D, Harder J, Zemlin M, Gomez de Agüero M, Masjosthusmann K, and Humberg A
Subjects: Humans, Infant, Newborn, Immunity, Innate, Animals, Dysbiosis microbiology, Infant, Premature, Microbiota, Antimicrobial Peptides
Abstract: Antimicrobial peptides (AMPs) are crucial components of the innate immune system in various organisms, including humans. Beyond their direct antimicrobial effects, AMPs play essential roles in various physiological processes. They induce angiogenesis, promote wound healing, modulate immune responses, and serve as chemoattractants for immune cells. AMPs regulate the microbiome and combat microbial infections on the skin, lungs, and gastrointestinal tract. Produced in response to microbial signals, AMPs help maintain a balanced microbial community and provide a first line of defense against infection. In preterm infants, alterations in microbiome composition have been linked to various health outcomes, including sepsis, necrotizing enterocolitis, atopic dermatitis, and respiratory infections. Dysbiosis, or an imbalance in the microbiome, can alter AMP profiles and potentially lead to inflammation-mediated diseases such as chronic lung disease and obesity. In the following review, we summarize what is known about the vital role of AMPs as multifunctional peptides in protecting newborn infants against infections and modulating the microbiome and immune response. Understanding their roles in preterm infants and high-risk populations offers the potential for innovative approaches to disease prevention and treatment.
Published: 2024
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30. Complex Arterio-Portal-Venous Malformation of the Liver.

Author: Wildgruber M, Köhler M, Masjosthusmann K, Hoffmann F, and Häberle B
Abstract: A neonatal female patient exhibited a congenital intricate vascular malformation affecting the liver, encompassing anomalies in the arterial, venous, and portal venous systems and notably including an aneurysm within the portal vein. The management strategy involved a staged endovascular approach, initially using retrograde embolization via the venous outflow tract. Subsequently, transarterial embolization was performed to address complications associated with pulmonary and portal hypertension., Competing Interests: The authors have reported that they have no relationships relevant to the contents of this paper to disclose., (© 2024 The Authors.)
Published: 2024
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31. Diagnostic evaluation of paediatric autoimmune lymphoproliferative immunodeficiencies (ALPID): a prospective cohort study.

Author: Hägele P, Staus P, Scheible R, Uhlmann A, Heeg M, Klemann C, Maccari ME, Ritterbusch H, Armstrong M, Cutcutache I, Elliott KS, von Bernuth H, Leahy TR, Leyh J, Holzinger D, Lehmberg K, Svec P, Masjosthusmann K, Hambleton S, Jakob M, Sparber-Sauer M, Kager L, Puzik A, Wolkewitz M, Lorenz MR, Schwarz K, Speckmann C, Rensing-Ehl A, and Ehl S
Subjects: Male, Female, Child, Humans, Child, Preschool, Adolescent, Prospective Studies, Biomarkers, Adaptor Proteins, Signal Transducing genetics, Autoimmune Lymphoproliferative Syndrome diagnosis, Autoimmune Lymphoproliferative Syndrome genetics, Autoimmune Lymphoproliferative Syndrome therapy, Common Variable Immunodeficiency, Anemia, Hemolytic, Autoimmune, Thrombocytopenia
Abstract: Background: Lymphoproliferation and autoimmune cytopenias characterise autoimmune lymphoproliferative syndrome. Other conditions sharing these manifestations have been termed autoimmune lymphoproliferative syndrome-like diseases, although they are frequently more severe. The aim of this study was to define the genetic, clinical, and immunological features of these disorders to improve their diagnostic classification., Methods: In this prospective cohort study, patients were referred to the Center for Chronic Immunodeficiency in Freiburg, Germany, between Jan 1, 2008 and March 5, 2022. We enrolled patients younger than 18 years with lymphoproliferation and autoimmune cytopenia, lymphoproliferation and at least one additional sign of an inborn error of immunity (SoIEI), bilineage autoimmune cytopenia, or autoimmune cytopenia and at least one additional SoIEI. Autoimmune lymphoproliferative syndrome biomarkers were determined in all patients. Sanger sequencing followed by in-depth genetic studies were recommended for patients with biomarkers indicative of autoimmune lymphoproliferative syndrome, while IEI panels, exome sequencing, or genome sequencing were recommended for patients without such biomarkers. Genetic analyses were done as decided by the treating physician. The study was registered on the German Clinical Trials Register, DRKS00011383, and is ongoing., Findings: We recruited 431 children referred for autoimmune lymphoproliferative syndrome evaluation, of whom 236 (55%) were included on the basis of lymphoproliferation and autoimmune cytopenia, 148 (34%) on the basis of lymphoproliferation and another SoIEI, 33 (8%) on the basis of autoimmune bicytopenia, and 14 (3%) on the basis of autoimmune cytopenia and another SoIEI. Median age at diagnostic evaluation was 9·8 years (IQR 5·5-13·8), and the cohort comprised 279 (65%) boys and 152 (35%) girls. After biomarker and genetic assessments, autoimmune lymphoproliferative syndrome was diagnosed in 71 (16%) patients. Among the remaining 360 patients, 54 (15%) had mostly autosomal-dominant autoimmune lymphoproliferative immunodeficiencies (AD-ALPID), most commonly affecting JAK-STAT (26 patients), CTLA4-LRBA (14), PI3K (six), RAS (five), or NFκB (three) signalling. 19 (5%) patients had other IEIs, 17 (5%) had non-IEI diagnoses, 79 (22%) were unresolved despite extended genetics (ALPID-U), and 191 (53%) had insufficient genetic workup for diagnosis. 16 (10%) of 161 patients with a final diagnosis had somatic mutations. Alternative classification of patients fulfilling common variable immunodeficiency or Evans syndrome criteria did not increase the proportion of genetic diagnoses., Interpretation: The ALPID phenotype defined in this study is enriched for patients with genetic diseases treatable with targeted therapies. The term ALPID might be useful to focus diagnostic and therapeutic efforts by triggering extended genetic analysis and consideration of targeted therapies, including in some children currently classified as having common variable immunodeficiency or Evans syndrome., Funding: Deutsche Forschungsgemeinschaft under Germany's Excellence Strategy., Translation: For the German translation of the abstract see Supplementary Materials section., Competing Interests: Declaration of interests SE are members of a data monitoring committee for leniolisib (Pharming). SH and SE received research funding from Pharming. MA and IC are a full-time employees of UCB Pharma and are shareholders of UCB. SE received research support from UCB for this study, including the whole-genome trio analysis of 30 families. All other authors declare no competing interests., (Copyright © 2024 Elsevier Ltd. All rights reserved.)
Published: 2024
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32. Type 1 Interferon-Stimulated Gene Expression and Disease Activity in Pediatric Rheumatic Diseases: No Composite Scores Needed?

Author: Weiden C, Saers M, Schwarz T, Hinze T, Wittkowski H, Kessel C, Masjosthusmann K, Mohr M, Evers G, Oesingmann-Weirich S, Foell D, and Hinze CH
Abstract: Objective: Rheumatic diseases are characterized by different patterns of immune overactivation. This study investigated the correlation of whole blood type 1 interferon (IFN) stimulated gene (ISG), IL18, and CXCL9 expression with clinical disease activity in pediatric rheumatic diseases and assessed the required number of ISGs to be included in a composite type 1 IFN score., Methods: Whole blood-derived RNA and clinical data were collected from 171 mostly pediatric patients with connective tissue diseases (CTDs), systemic autoinflammatory diseases (SAIDs), monogenic interferonopathies (IFNPs) and other inflammatory diseases, and from 38 controls. The expression of six previously established ISGs, IL18, and CXCL9 was assessed by real-time polymerase chain reaction (471 samples). Individual and composite gene expression was assessed, and correlation and threshold analyses were performed., Results: Correlation between ISG expression and clinical disease activity was strongest in CTD, especially in juvenile dermatomyositis (JDM) and IFNP, and modest in patients with SAID. Threshold ISG expression levels for the detection of at least mild clinical disease activity were substantially higher in patients with systemic lupus erythematosus compared with JDM. The correlation of expression levels of limited sets of ISGs and even individual ISGs with clinical disease activity were not inferior to a composite score of six ISGs., Conclusion: In a real-world cohort, individual ISG expression levels robustly reflected clinical disease activity in CTD and IFNP, especially in JDM, which would simplify such analyses in clinical routine and be more cost-effective. Threshold levels varied across diseases, potentially reflecting different mechanisms of type 1 IFN overactivation., (© 2023 The Authors. ACR Open Rheumatology published by Wiley Periodicals LLC on behalf of American College of Rheumatology.)
Published: 2023
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33. Developing a Method for Quantifying Meropenem in Children-Volumetric Adsorptive Microsampling Versus Plasma Sampling.

Author: Ramadan O, Schatz LM, van den Heuvel I, Masjosthusmann K, Groll AH, and Hempel G
Subjects: Humans, Child, Meropenem, Chromatography, High Pressure Liquid methods, Anti-Bacterial Agents, Drug Monitoring methods, Dried Blood Spot Testing methods, Blood Specimen Collection methods, Tandem Mass Spectrometry methods
Abstract: Background: Meropenem is a carbapenem antibiotic often used in pediatric intensive care units due to its broad spectrum of activity. Therapeutic drug monitoring (TDM) is a useful tool to increase the effectiveness of meropenem by adjusting the dose based on plasma levels; however, the relatively large sample volume required for TDM can limit its use in children. Therefore, this study aimed to determine meropenem concentrations and consequently perform TDM effectively using the smallest possible sample volume. Volumetric absorptive microsampling (VAMS) is a sampling technology developed to collect a small, precise volume of blood. For the applicability of VAMS in TDM, plasma concentrations must be reliably calculated from whole blood (WB) collected by VAMS., Methods: VAMS technology using 10 µL of WB was evaluated and compared with EDTA-plasma sampling. High-performance liquid chromatography with UV detection was applied to quantify meropenem in VAMS and plasma samples after the removal of proteins by precipitation. Ertapenem was used as the internal standard. Samples were collected simultaneously from critically ill children receiving meropenem using VAMS and traditional sampling., Results: It was found that no consistent factor could be determined to calculate meropenem plasma concentrations from the WB, indicating that VAMS was not reliable in the TDM of meropenem. Therefore, to reduce the required sample amount in pediatric patients, a method for quantifying meropenem from 50 µL of plasma with a lower limit of quantification of 1 mg/L was developed and successfully validated., Conclusions: A simple, reliable, and low-cost method was established using high-performance liquid chromatography-UV to determine the concentration of meropenem in 50 µL of plasma. VAMS using WB does not seem to be suitable for TDM of meropenem., Competing Interests: The authors declare no conflict of interest., (Copyright © 2023 Wolters Kluwer Health, Inc. All rights reserved.)
Published: 2023
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34. Continuous Kidney Replacement Therapy Practices in Pediatric Intensive Care Units Across Europe.

Author: Daverio M, Cortina G, Jones A, Ricci Z, Demirkol D, Raymakers-Janssen P, Lion F, Camilo C, Stojanovic V, Grazioli S, Zaoral T, Masjosthusmann K, Vankessel I, and Deep A
Subjects: Infant, Newborn, Child, Humans, Cross-Sectional Studies, Heparin, Intensive Care Units, Pediatric, Europe, Diuretics, Continuous Renal Replacement Therapy
Abstract: Importance: Continuous kidney replacement therapy (CKRT) is the preferred method of kidney support for children with critical illness in pediatric intensive care units (PICUs). However, there are no data on the current CKRT management practices in European PICUs., Objective: To describe current CKRT practices across European PICUs., Design, Setting, and Participants: This cross-sectional survey of PICUs in 20 European countries was conducted by the Critical Care Nephrology Section of the European Society of Pediatric and Neonatal Intensive Care from April 1, 2020, to May 31, 2022. Participants included intensivists and nurses working in European PICUs. The survey was developed in English and distributed using SurveyMonkey. One response from each PICU that provided CKRT was included in the analysis. Data were analyzed from June 1 to June 30, 2022., Main Outcome and Measures: Demographic characteristics of European PICUs along with organizational and delivery aspects of CKRT (including prescription, liberation from CKRT, and training and education) were assessed., Results: Of 283 survey responses received, 161 were included in the analysis (response rate, 76%). The attending PICU consultant (70%) and the PICU team (77%) were mainly responsible for CKRT prescription, whereas the PICU nurses were responsible for circuit setup (49%) and bedside machine running (67%). Sixty-one percent of permanent nurses received training to use CKRT, with no need for certification or recertification in 36% of PICUs. Continuous venovenous hemodiafiltration was the preferred dialytic modality (51%). Circuit priming was performed with normal saline (67%) and blood priming in children weighing less than 10 kg (56%). Median (IQR) CKRT dose was 35 (30-50) mL/kg/h in neonates and 30 (30-40) mL/kg/h in children aged 1 month to 18 years. Forty-one percent of PICUs used regional unfractionated heparin infusion, whereas 35% used citrate-based regional anticoagulation. Filters were changed for filter clotting (53%) and increased transmembrane pressure (47%). For routine circuit changes, 72 hours was the cutoff in 62% of PICUs. Some PICUs (34%) monitored fluid removal goals every 4 hours, with variation from 12 hours (17%) to 24 hours (13%). Fluid removal goals ranged from 1 to 3 mL/kg/h. Liberation from CKRT was performed with a diuretic bolus followed by an infusion (32%) or a diuretic bolus alone (19%)., Conclusions and Relevance: This survey study found a wide variation in current CKRT practice, including organizational aspects, education and training, prescription, and liberation from CKRT, in European PICUs. This finding calls for concerted efforts on the part of the pediatric critical care and nephrology communities to streamline CKRT education and training, research, and guidelines to reduce variation in practice.
Published: 2022
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35. An Immunological Axis Involving Interleukin 1β and Leucine-Rich-α2-Glycoprotein Reflects Therapeutic Response of Children with Kawasaki Disease: Implications from the KAWAKINRA Trial.

Author: Kessel C, Koné-Paut I, Tellier S, Belot A, Masjosthusmann K, Wittkowski H, Fuehner S, Rossi-Semerano L, Dusser P, Marie I, Boukhedouni N, Agostini H, Piedvache C, and Foell D
Subjects: Biomarkers, Child, Endothelial Cells metabolism, Glycoproteins metabolism, Glycoproteins therapeutic use, Humans, Immunoglobulins, Intravenous therapeutic use, Interleukin 1 Receptor Antagonist Protein therapeutic use, Interleukin-1beta, Interleukin-6 metabolism, Leucine therapeutic use, Retrospective Studies, S100A12 Protein, Mucocutaneous Lymph Node Syndrome complications, Mucocutaneous Lymph Node Syndrome drug therapy
Abstract: Purpose: A recent phase II open-label study of the interleukin 1 (IL-1) receptor antagonist (IL-1Ra) anakinra in treating IVIG-resistant Kawasaki disease (KD) patients reported promising results. Here, we aimed to characterize the immunological impact of IL-1 blockade in this unique study population., Methods: Patients' and control sera and supernatants of cells (whole blood, neutrophils, coronary artery endothelial cells) stimulated with recombinant IL-1β were analyzed for single or multiple marker (n = 22) expression by ELISA or multiplexed bead array assay. Data were analyzed using unsupervised hierarchical clustering, multiple correlation, and multi-comparison statistics and were compared to retrospective analyses of KD transcriptomics., Results: Inflammation in IVIG-resistant KD (n = 16) is hallmarked by over-expression of innate immune mediators (particularly IL-6 > CXCL10 > S100A12 > IL-1Ra). Those as well as levels of immune or endothelial cell activation markers (sICAM-1, sVCAM-1) declined most significantly in course of anakinra treatment. Prior as well as following IL-1R blockade, over-expression of leucine-rich-α2-glycoprotein 1 (LRG1) associated best with remnant inflammatory activity and the necessity to escalate anakinra dosage and separated inflammatory KD patients from sJIA-MAS (n = 13) and MIS-C (n = 4). Protein as well as retrospective gene expression analyses indicated tight association of LRG1 with IL-1β signaling and neutrophilia, while particularly neutrophil stimulation with recombinant IL-1β resulted in concentration-dependent LRG1 release., Conclusion: Our study identifies LRG1 as known trigger of endothelial activation and cardiac re-modeling to associate with IL-1β signaling in KD. Besides a potential patho-mechanistic implication of these findings, our data suggest blood leukocyte and neutrophil counts to best predict response to IL-1Ra treatment in IVIG-resistant KD., (© 2022. The Author(s).)
Published: 2022
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36. Autoantibodies against interleukin-1 receptor antagonist in multisystem inflammatory syndrome in children: a multicentre, retrospective, cohort study.

Author: Pfeifer J, Thurner B, Kessel C, Fadle N, Kheiroddin P, Regitz E, Hoffmann MC, Kos IA, Preuss KD, Fischer Y, Roemer K, Lohse S, Heyne K, Detemple MC, Fedlmeier M, Juenger H, Sauer H, Meyer S, Rohrer T, Wittkowski H, Becker SL, Masjosthusmann K, Bals R, Gerling S, Smola S, Bewarder M, Birk E, Keren A, Böhm M, Jakob A, Abdul-Khaliq H, Anton J, Kabesch M, Pino-Ramirez RM, Foell D, and Thurner L
Abstract: Background: Multisystem inflammatory syndrome in children (MIS-C) is a rare but serious complication of infection with SARS-CoV-2. A possible involvement of pathogenetically relevant autoantibodies has been discussed. Recently, neutralising autoantibodies against inflammatory receptor antagonists progranulin and interleukin-1 receptor antagonist (IL-1Ra) were found in adult patients with critical COVID-19. The aim of this study was to investigate the role of such autoantibodies in MIS-C., Methods: In this multicentre, retrospective, cohort study, plasma and serum samples were collected from patients (0-18 years) with MIS-C (as per WHO criteria) treated at five clinical centres in Germany and Spain. As controls, we included plasma or serum samples from children with Kawasaki disease, children with inactive systemic juvenile idiopathic arthritis, and children with suspected growth retardation (non-inflammatory control) across four clinical centres in Germany and Spain (all aged ≤18 years). Serum samples from the CoKiBa trial were used as two further control groups, from healthy children (negative for SARS-CoV-2 antibodies) and children with previous mild or asymptomatic COVID-19 (aged ≤17 years). MIS-C and control samples were analysed for autoantibodies against IL-1Ra and progranulin, and for IL-1Ra concentrations, by ELISA. Biochemical analysis of plasma IL-1Ra was performed with native Western blots and isoelectric focusing. Functional activity of the autoantibodies was examined by an in vitro IL-1β-signalling reporter assay., Findings: Serum and plasma samples were collected between March 6, 2011, and June 2, 2021. Autoantibodies against IL-1Ra could be detected in 13 (62%) of 21 patients with MIS-C (11 girls and ten boys), but not in children with Kawasaki disease (n=24; nine girls and 15 boys), asymptomatic or mild COVID-19 (n=146; 72 girls and 74 boys), inactive systemic juvenile idiopathic arthritis (n=10; five girls and five boys), suspected growth retardation (n=33; 13 girls and 20 boys), or in healthy controls (n=462; 230 girls and 232 boys). Anti-IL-1Ra antibodies in patients with MIS-C belonged exclusively to the IgG1 subclass, except in one patient who had additional IL-1Ra-specific IgM antibodies. Autoantibodies against progranulin were only detected in one (5%) patient with MIS-C. In patients with MIS-C who were positive for anti-IL-1Ra antibodies, free plasma IL-1Ra concentrations were reduced, and immune-complexes of IL-1Ra were detected. Notably, an additional, hyperphosphorylated, transiently occurring atypical isoform of IL-1Ra was observed in all patients with MIS-C who were positive for anti-IL-1Ra antibodies. Anti-IL-1Ra antibodies impaired IL-1Ra function in reporter cell assays, resulting in amplified IL-1β signalling., Interpretation: Anti-IL-1Ra autoantibodies were observed in a high proportion of patients with MIS-C and were specific to these patients. Generation of these autoantibodies might be triggered by an atypical, hyperphosphorylated isoform of IL-1Ra. These autoantibodies impair IL-1Ra bioactivity and might thus contribute to increased IL-1β-signalling in MIS-C., Funding: NanoBioMed fund of the University of Saarland, José Carreras Center for Immuno and Gene Therapy, Dr Rolf M Schwiete Stiftung, Staatskanzlei Saarland, German Heart Foundation, Charity of the Blue Sisters, Bavarian Ministry of Health, the Center for Interdisciplinary Clinical Research at University Hospital Münster, EU Horizon 2020., Competing Interests: LT has received research grants from Wilhelm Sander-Stiftung, BioNanoMed, and the Homburger Forschungsförderung programme of the University of Saarland; travel grants for meeting attendance from AbbVie, Janssen, and EUSA-Pharm; and has participated in advisory boards for Takeda, AstraZeneca, Merck, and EUSA Pharma. LT and K-DP were listed among inventors of a patent on progranulin antibodies as marker for autoimmune diseases filed by University of Saarland, which expired in 2017. CK has received consulting fees from Novartis and Swedish Orphan Biovitrum (SOBI) and receives research support from Novartis. MBö has received speakers honoraria from or participated in advisory boards for Abbott, Amgen, AstraZeneca, Bayer, Boehringer-Ingelheim, Bristol Myers Squibb, Cyotkinetics, Medtronic, Novartis, Servier, and Vifor. HW has received honoraria (lecture fees) from Novartis and Takeda, and travel support from Octapharma and CSL-Behring. DF has received speaker fees or honoraria from Chugai-Roche, Novartis, and SOBI, and research support from Novartis, Pfizer, and SOBI. JA has received grants and travel grants for meeting attendance from SOBI and Novartis, and participated in advisory boards for SOBI and Novartis. BT has received honoraria for lectures from Nutricia Milupa and is a private shareholder of BioNTech. MK has received research support for the CoKiBa trial from Roche, who provided the diagnostic antibodies at a time when they were not yet commercially available. In addition, MK has received honoraria for lectures from Pädnetz Bayern, Ärztlicher Kreisverband, and Bayerischer Berufsverband der Kinder- und Jugendärzte, and participated in the advisory board for COVID-19 in children of the Bavarian Ministry of Health. SLB has participated in advisory boards and received honoraria for lectures from Shinogi and Pfizer. RB has received grants and personal fees from AstraZeneca, Boehringer Ingelheim, and Novartis; personal fees from GlaxoSmithKline, Grifols, and CSL Behring; grants from the German Ministry of Education and Research, Competence Network on Asthma and COPD, Sander Stiftung, Schwiete Stiftung, Krebshilfe, and Mukosviszidose; and has a leadership role at the Alpha-1-Center in Bad Lippspringe, Germany. All other authors declare no competing interests., (© 2022 Elsevier Ltd. All rights reserved.)
Published: 2022
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37. Stenotrophomonas maltophilia Infections in Pediatric Patients - Experience at a European Center for Pediatric Hematology and Oncology.

Author: Zöllner SK, Kampmeier S, Froböse NJ, Herbrüggen H, Masjosthusmann K, van den Heuvel A, Reicherts C, Ranft A, and Groll AH
Abstract: Stenotrophomonas maltophilia is an important nosocomial pathogen in immunocom-promised individuals and characterized by intrinsic resistance to broad-spectrum antibacterial agents. Limited data exists on its clinical relevance in immunocompromised pediatric patients, particularly those with hematological or oncological disorders. In a retrospective single center cohort study in pediatric patients receiving care at a large european pediatric hematology and oncology department, ten cases of invasive S.maltophilia infections (blood stream infections (BSI), 4; BSI and pneumonia, 3, or soft tissue infection, 2; and pneumonia, 1) were identified between 2010 and 2020. Seven patients had lymphoblastic leukemia and/or were post allogeneic hematopoietic cell transplantation. Invasive S.maltophilia infections occurred in a setting of indwelling central venous catheters, granulocytopenia, defective mucocutaneous barriers, treatment with broad-spectrum antibacterial agents, and admission to the intensive care unit. Whole genome sequencing based typing revealed no genetic relationship among four individual S.maltophilia isolates. The case fatality rate and mortality at 100 days post diagnosis were 40 and 50%, respectively, and three patients died from pulmonary hemorrhage. Invasive S.maltophilia infections are an emerging cause of infectious morbidity in patients receiving care at departments of pediatric hematology and oncology and carry a high case fatality rate., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2021 Zöllner, Kampmeier, Froböse, Herbrüggen, Masjosthusmann, van den Heuvel, Reicherts, Ranft and Groll.)
Published: 2021
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38. Discrimination of COVID-19 From Inflammation-Induced Cytokine Storm Syndromes Using Disease-Related Blood Biomarkers.

Author: Kessel C, Vollenberg R, Masjosthusmann K, Hinze C, Wittkowski H, Debaugnies F, Nagant C, Corazza F, Vély F, Kaplanski G, Girard-Guyonvarc'h C, Gabay C, Schmidt H, Foell D, and Tepasse PR
Subjects: Adolescent, Adult, Aged, Aged, 80 and over, Biomarkers blood, COVID-19 blood, COVID-19 complications, Cytokine Release Syndrome blood, Diagnosis, Differential, Female, Humans, Lymphohistiocytosis, Hemophagocytic blood, Lymphohistiocytosis, Hemophagocytic complications, Macrophage Activation Syndrome blood, Macrophage Activation Syndrome complications, Male, Middle Aged, Young Adult, COVID-19 diagnosis, Cytokine Release Syndrome etiology, Interleukin-18 blood, Interleukin-8 blood, Lymphohistiocytosis, Hemophagocytic diagnosis, Macrophage Activation Syndrome diagnosis
Abstract: Objective: Infection with the novel coronavirus SARS-CoV-2 triggers severe illness with high mortality in a subgroup of patients. Such a critical course of COVID-19 is thought to be associated with the development of cytokine storm, a condition seen in macrophage activation syndrome (MAS) and secondary hemophagocytic lymphohistiocytosis (HLH). However, specific data demonstrating a clear association of cytokine storm with severe COVID-19 are still lacking. The aim of this study was to directly address whether immune activation in COVID-19 does indeed mimic the conditions found in these classic cytokine storm syndromes., Methods: Levels of 22 biomarkers were quantified in serum samples from patients with COVID-19 (n = 30 patients, n = 83 longitudinal samples in total), patients with secondary HLH/MAS (n = 50), and healthy controls (n = 9). Measurements were performed using bead array assays and single-marker enzyme-linked immunosorbent assay. Serum biomarker levels were assessed for correlations with disease outcome., Results: In patients with secondary HLH/MAS, we observed pronounced activation of the interleukin-18 (IL-18)-interferon-γ axis, increased serum levels of IL-1 receptor antagonist, intercellular adhesion molecule 1, and IL-8, and strongly reduced levels of soluble Fas ligand in the course of SARS-CoV-2 infection. These observations appeared to discriminate immune dysregulation in critical COVID-19 from the well-recognized characteristics of other cytokine storm syndromes., Conclusion: Serum biomarker profiles clearly separate COVID-19 from MAS or secondary HLH in terms of distinguishing the severe systemic hyperinflammation that occurs following SARS-CoV-2 infection. These findings could be useful in determining the efficacy of drugs targeting key molecules and pathways specifically associated with systemic cytokine storm conditions in the treatment of COVID-19., (© 2021 The Authors. Arthritis & Rheumatology published by Wiley Periodicals LLC on behalf of American College of Rheumatology.)
Published: 2021
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39. Extracorporeal Membrane Oxygenation in Children With Cancer or Hematopoietic Cell Transplantation: Single-Center Experience in 20 Consecutive Patients.

Author: Potratz JC, Guddorf S, Ahlmann M, Tekaat M, Rossig C, Omran H, Masjosthusmann K, and Groll AH
Abstract: Extracorporeal membrane oxygenation (ECMO) is a rescue therapy for severe respiratory and/or circulatory failure. Few data exist on the potential benefit of ECMO in immunocompromised pediatric patients with cancer and/or hematopoietic cell transplantation (HCT). Over a period of 12 years, eleven (1.9%) of 572 patients with new diagnosis of leukemia/lymphoma and nine (3.5%) of 257 patients post allogeneic HCT underwent ECMO at our center. Five (45%) and two (22%) patients, respectively, survived to hospital discharge with a median event-free survival of 4.2 years. Experiences and outcomes in this cohort may aid clinicians and families when considering ECMO for individual patients., Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2021 Potratz, Guddorf, Ahlmann, Tekaat, Rossig, Omran, Masjosthusmann and Groll.)
Published: 2021
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40. Treatment of Children With Cancer and/or Hematopoietic Stem Cell Transplantation in the Intensive Care Unit: Experience at a Large European Pediatric Cancer Center.

Author: Barking CTMM, Masjosthusmann K, Rellensmann G, Ehlert K, Zöllner S, Jocham S, Kremer A, Potratz J, Rieger-Fackeldey E, and Groll AH
Subjects: Adolescent, Child, Child, Preschool, Europe, Female, Hematopoietic Stem Cell Transplantation mortality, Humans, Intensive Care Units statistics & numerical data, Male, Neoplasms mortality, Retrospective Studies, Hematopoietic Stem Cell Transplantation adverse effects, Neoplasms complications, Neoplasms therapy
Abstract: Pediatric cancer treatment and hematopoietic stem cell transplantation (HSCT) carry considerable risks of morbidity. We conducted a single-center retrospective analysis of intensive care unit (ICU) admissions in unselected children and adolescents treated for cancer or undergoing HSCT. In a 10-year time period, 140 patients had 188 ICU admissions for a life-threatening condition. Main reasons for ICU admission were respiratory or cardiovascular insufficiency and sepsis. Mortality in the ICU was 19.1% and related to organ failure or acute complications in 77.8% and progress of the underlying malignancy in 22.2%. Mortality rates at 30, 100, and 365 days after discharge from the ICU were 24.5%, 30.9%, and 39.9%. Kaplan-Meier survival probabilities at 5 and 10 years were 46.4% and 39.8%, respectively. Multivariable analysis revealed the number of failed organ systems, the number of prior ICU stays, and days spent in the ICU as parameters independently associated with death. Taken together, the outcome of pediatric cancer and/or HSCT patients admitted to the ICU for life-threatening conditions was not as dismal as reported elsewhere. Most patients benefitted from ICU care, and survival was predominantly compromised by the evolution of complications.
Published: 2020
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41. Application of two different nasal CPAP levels for the treatment of respiratory distress syndrome in preterm infants-"The OPTTIMMAL-Trial"-Optimizing PEEP To The IMMAture Lungs: study protocol of a randomized controlled trial.

Author: Waitz M, Engel C, Schloesser R, Rochwalsky U, Meyer S, Larsen A, Hoffmann P, Zemlin M, Bohnhorst B, Peter C, Hoppenz M, Pabst T, Zimmer KP, Franz AR, Haertel C, Frieauff E, Sandkötter J, Masjosthusmann K, Deindl P, Singer D, Heidkamp M, Schmidt A, and Ehrhardt H
Subjects: Continuous Positive Airway Pressure, Humans, Infant, Infant, Newborn, Infant, Premature, Lung, Multicenter Studies as Topic, Randomized Controlled Trials as Topic, Resuscitation, Respiratory Distress Syndrome, Respiratory Distress Syndrome, Newborn diagnosis, Respiratory Distress Syndrome, Newborn therapy
Abstract: Background: Nasal continuous positive airway pressure (CPAP) applies positive end-expiratory pressure (PEEP) and has been shown to reduce the need for intubation and invasive mechanical ventilation in very low birth weight infants with respiratory distress syndrome. However, CPAP failure rates of 50% are reported in large randomized controlled trials. A possible explanation for these failure rates is the application of insufficient low levels of PEEP during nasal CPAP treatment to maintain adequate functional residual capacity shortly after birth. The optimum PEEP level to treat symptoms of respiratory distress in very low birth weight infants has not been assessed in clinical studies. The aim of the study is to compare two different PEEP levels during nasal CPAP treatment in preterm infants., Methods: In this randomized multicenter trial, 216 preterm infants born at 26 + 0-29 + 6 gestational weeks will be allocated to receive a higher (6-8 cmH 2 O) or a lower (3-5 cmH 2 O) PEEP during neonatal resuscitation and the first 120 h of life. The PEEP level within each group will be titrated throughout the intervention based on the FiO 2 (fraction of inspired oxygen concentration) requirements to keep oxygenation within the target range. The primary outcome is defined as the need for intubation and mechanical ventilation for > 1 h or being not ventilated but reaching one of the two pre-defined CPAP failure criteria (FiO 2 > 0.5 for > 1 h or pCO 2 ≥ 70 mmHg in two consecutive blood gas analyses at least 2 h apart)., Discussion: Based on available data from the literature, the optimum level of PEEP that most effectively treats respiratory distress syndrome in preterm infants is unknown, since the majority of large clinical trials applied a wide range of PEEP levels (4-8 cmH 2 O). The rationale for our study hypothesis is that the early application of a higher PEEP level will more effectively counteract the collapsing properties of the immature and surfactant-deficient lungs and that the level of inspired oxygen may serve as a surrogate marker to guide PEEP titration. Finding the optimum noninvasive continuous distending pressure during early nasal CPAP is required to improve CPAP efficacy and as a consequence to reduce the exposure to ventilator-induced lung injury and the incidence of chronic lung disease in this vulnerable population of very preterm infants., Trial Registration: drks.de DRKS00019940 . Registered on March 13, 2020.
Published: 2020
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42. The German National Registry of Primary Immunodeficiencies (2012-2017).

Author: El-Helou SM, Biegner AK, Bode S, Ehl SR, Heeg M, Maccari ME, Ritterbusch H, Speckmann C, Rusch S, Scheible R, Warnatz K, Atschekzei F, Beider R, Ernst D, Gerschmann S, Jablonka A, Mielke G, Schmidt RE, Schürmann G, Sogkas G, Baumann UH, Klemann C, Viemann D, von Bernuth H, Krüger R, Hanitsch LG, Scheibenbogen CM, Wittke K, Albert MH, Eichinger A, Hauck F, Klein C, Rack-Hoch A, Sollinger FM, Avila A, Borte M, Borte S, Fasshauer M, Hauenherm A, Kellner N, Müller AH, Ülzen A, Bader P, Bakhtiar S, Lee JY, Heß U, Schubert R, Wölke S, Zielen S, Ghosh S, Laws HJ, Neubert J, Oommen PT, Hönig M, Schulz A, Steinmann S, Schwarz K, Dückers G, Lamers B, Langemeyer V, Niehues T, Shai S, Graf D, Müglich C, Schmalzing MT, Schwaneck EC, Tony HP, Dirks J, Haase G, Liese JG, Morbach H, Foell D, Hellige A, Wittkowski H, Masjosthusmann K, Mohr M, Geberzahn L, Hedrich CM, Müller C, Rösen-Wolff A, Roesler J, Zimmermann A, Behrends U, Rieber N, Schauer U, Handgretinger R, Holzer U, Henes J, Kanz L, Boesecke C, Rockstroh JK, Schwarze-Zander C, Wasmuth JC, Dilloo D, Hülsmann B, Schönberger S, Schreiber S, Zeuner R, Ankermann T, von Bismarck P, Huppertz HI, Kaiser-Labusch P, Greil J, Jakoby D, Kulozik AE, Metzler M, Naumann-Bartsch N, Sobik B, Graf N, Heine S, Kobbe R, Lehmberg K, Müller I, Herrmann F, Horneff G, Klein A, Peitz J, Schmidt N, Bielack S, Groß-Wieltsch U, Classen CF, Klasen J, Deutz P, Kamitz D, Lassay L, Tenbrock K, Wagner N, Bernbeck B, Brummel B, Lara-Villacanas E, Münstermann E, Schneider DT, Tietsch N, Westkemper M, Weiß M, Kramm C, Kühnle I, Kullmann S, Girschick H, Specker C, Vinnemeier-Laubenthal E, Haenicke H, Schulz C, Schweigerer L, Müller TG, Stiefel M, Belohradsky BH, Soetedjo V, Kindle G, and Grimbacher B
Subjects: Adolescent, Adult, Aged, Aged, 80 and over, Child, Child, Preschool, Delayed Diagnosis, Female, Genetic Therapy, Germany epidemiology, Hematopoietic Stem Cell Transplantation, Humans, Immunoglobulins therapeutic use, Infant, Infant, Newborn, Male, Middle Aged, Prevalence, Registries, Young Adult, Immunologic Deficiency Syndromes diagnosis, Immunologic Deficiency Syndromes epidemiology, Immunologic Deficiency Syndromes genetics, Immunologic Deficiency Syndromes therapy
Abstract: Introduction: The German PID-NET registry was founded in 2009, serving as the first national registry of patients with primary immunodeficiencies (PID) in Germany. It is part of the European Society for Immunodeficiencies (ESID) registry. The primary purpose of the registry is to gather data on the epidemiology, diagnostic delay, diagnosis, and treatment of PIDs. Methods: Clinical and laboratory data was collected from 2,453 patients from 36 German PID centres in an online registry. Data was analysed with the software Stata® and Excel. Results: The minimum prevalence of PID in Germany is 2.72 per 100,000 inhabitants. Among patients aged 1-25, there was a clear predominance of males. The median age of living patients ranged between 7 and 40 years, depending on the respective PID. Predominantly antibody disorders were the most prevalent group with 57% of all 2,453 PID patients (including 728 CVID patients). A gene defect was identified in 36% of patients. Familial cases were observed in 21% of patients. The age of onset for presenting symptoms ranged from birth to late adulthood (range 0-88 years). Presenting symptoms comprised infections (74%) and immune dysregulation (22%). Ninety-three patients were diagnosed without prior clinical symptoms. Regarding the general and clinical diagnostic delay, no PID had undergone a slight decrease within the last decade. However, both, SCID and hyper IgE- syndrome showed a substantial improvement in shortening the time between onset of symptoms and genetic diagnosis. Regarding treatment, 49% of all patients received immunoglobulin G (IgG) substitution (70%-subcutaneous; 29%-intravenous; 1%-unknown). Three-hundred patients underwent at least one hematopoietic stem cell transplantation (HSCT). Five patients had gene therapy. Conclusion: The German PID-NET registry is a precious tool for physicians, researchers, the pharmaceutical industry, politicians, and ultimately the patients, for whom the outcomes will eventually lead to a more timely diagnosis and better treatment.
Published: 2019
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43. Successful Extracorporeal Life Support in a Pediatric Hematopoietic Stem Cell Transplant Recipient With Periengraftment Respiratory Failure.

Author: Potratz J, Ahlmann M, Rössig C, Omran H, and Masjosthusmann K
Subjects: Acute Disease, Adolescent, Allografts, Humans, Male, Neutropenia blood, Neutropenia etiology, Neutropenia therapy, Respiratory Insufficiency blood, Respiratory Insufficiency etiology, Extracorporeal Membrane Oxygenation, Graft Survival, Hematopoietic Stem Cell Transplantation, Respiratory Insufficiency therapy
Abstract: The use of extracorporeal life support (ECLS) as ultimate salvage therapy for hematopoietic stem cell transplant recipients remains controversial among oncologists and critical care specialists. Prognosis is poor, particularly after allogeneic transplantation, and literature to guide clinical decision-making is scarce. Our report describes successful ECLS in a pediatric patient undergoing allogeneic hematopoietic stem cell transplantation, who developed acute respiratory failure during severe neutropenia, followed by immediate neutrophil engraftment. This unique case highlights periengraftment respiratory failure as a possible patient subgroup that could benefit from ECLS; and illustrates that the distinct etiologies of respiratory failure and the patients' immune status deserve closer consideration in future studies evaluating ECLS in this high-risk population.
Published: 2018
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44. Munchausen by proxy syndrome mimicking systemic autoinflammatory disease: case report and review of the literature.

Author: Wittkowski H, Hinze C, Häfner-Harms S, Oji V, Masjosthusmann K, Monninger M, Grenzebach U, and Foell D
Subjects: Child, Preschool, Diagnosis, Differential, Humans, Male, Hereditary Autoinflammatory Diseases diagnosis, Munchausen Syndrome by Proxy diagnosis
Abstract: Background: Systemic autoinflammatory diseases (SAIDs) represent a growing number of monogenic, polygenic or multifactorial disorders that are often difficult to diagnose., Case Presentation: Here we report a patient who was initially erroneously diagnosed and treated for SAID. Symptoms consisted of recurrent fever, erythematous and/or blistering skin lesions, angioedema, susceptibility to bleeding, external ear infections and reversible anisocoria in the absence of laboratory evidence of systemic inflammation. After two and a half years of extensive diagnostic work-up and multiple empirical therapies, a final diagnosis of Munchausen by proxy syndrome (MBPS) was established., Conclusions: The diagnosis of SAID needs to be carefully reassessed if measurable systemic inflammation is missing, and MBPS should be included in the differential diagnosis.
Published: 2017
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45. Correlation of Secretory Activity of Neutrophils With Genotype in Patients With Familial Mediterranean Fever.

Author: Gohar F, Orak B, Kallinich T, Jeske M, Lieber M, von Bernuth H, Giese A, Weissbarth-Riedel E, Haas JP, Dressler F, Holzinger D, Lohse P, Neudorf U, Lainka E, Hinze C, Masjosthusmann K, Kessel C, Weinhage T, Foell D, and Wittkowski H
Subjects: Adolescent, Adult, Case-Control Studies, Caspase 1 blood, Child, Child, Preschool, Familial Mediterranean Fever blood, Familial Mediterranean Fever immunology, Female, Genotype, Heterozygote, Homozygote, Humans, In Vitro Techniques, Interleukin-18 blood, Interleukin-1beta blood, Male, Middle Aged, Neutrophils immunology, S100A12 Protein blood, Young Adult, Caspase 1 metabolism, Familial Mediterranean Fever genetics, Interleukin-18 metabolism, Interleukin-1beta metabolism, Neutrophils metabolism, Pyrin genetics, S100A12 Protein metabolism
Abstract: Objective: Familial Mediterranean fever (FMF) is an autoinflammatory disorder caused by pyrin-encoding MEFV mutations. Patients present with recurrent but self-limiting episodes of acute inflammation and often have persistent subclinical inflammation. The pathophysiology is only partially understood, but neutrophil overactivation is a hallmark of the disease. S100A12 is a neutrophil-derived proinflammatory danger signal that is strongly elevated in active FMF. This study was undertaken to characterize the secretory activity of neutrophils in vitro and investigate the association of S100A12 with disease activity and genotype in patients with FMF., Methods: Neutrophils from FMF patients carrying the p.M694V mutation (1 compound heterozygous and 5 homozygous) and neutrophils from 4 healthy control subjects were purified and stimulated in vitro. Neutrophil secretion of S100A12, interleukin-18 (IL-18), IL-1β, and caspase 1 was determined. Based on these in vitro analyses, serum concentrations of S100A12, IL-18, and IL-1β were also analyzed in 128 clinically and genetically characterized patients with FMF., Results: In vitro, unstimulated neutrophils from p.M694V-positive patients spontaneously secreted more S100A12, IL-18, and caspase 1 compared to neutrophils from healthy controls. Serum concentrations of S100A12 correlated with disease activity and genotype, with the levels being highest in homozygous patients and with compound heterozygotes displaying higher levels than heterozygotes. Compared to individuals negative for the p.M694V mutation, heterozygous, compound heterozygous, or homozygous p.M694V-positive patients had higher serum levels of S100A12 and IL-18 during inactive and subclinical disease., Conclusion: The FMF phenotype is known to be more severe in patients carrying the p.M694V mutation. This report describes 2 molecules secreted by unconventional secretory pathways, S100A12 and IL-18, whose concentrations correlated with clinical disease activity and genotype in patients with FMF. In this clinically and genetically heterogeneous disease, management of these surrogate markers might help to improve patient care and outcomes., (© 2016, American College of Rheumatology.)
Published: 2016
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46. Renal oligo- and anhydramnios: cause, course and outcome--a single-center study.

Author: Spiro JE, Konrad M, Rieger-Fackeldey E, Masjosthusmann K, Amler S, Klockenbusch W, and Schmitz R
Subjects: Abnormalities, Multiple diagnostic imaging, Adult, Congenital Abnormalities, Female, Humans, Kidney abnormalities, Kidney Diseases congenital, Kidney Tubules, Proximal abnormalities, Male, Pregnancy, Pregnancy Complications, Pregnancy Outcome, Prenatal Diagnosis adverse effects, Prognosis, Retrospective Studies, Urogenital Abnormalities, Hydronephrosis etiology, Oligohydramnios etiology, Prenatal Diagnosis methods, Ultrasonography, Prenatal methods
Abstract: Purpose: To evaluate the outcome of patients with renal oligohydramnios and explore the impact of patient variables on outcome and prognosis., Methods: A retrospective single-center study was conducted analyzing 104 pregnancies complicated by oligohydramnios of renal origin from 2001 to 2011. Statistical analysis was performed to assess the influence of pre- and postnatal data on pregnancy outcome, morbidity and mortality., Results: Prenatal renal diagnoses were as follows: hydronephrosis/megaureter: n = 21 (20.2 %), aberrance in renal form or location: n = 7 (6.7 %), cystic renal disease: n = 28 (26.9 %), renal dysplasia: n = 24 (23.1 %), renal agenesis: n = 42 (30.4 %), posterior urethral valves: n = 11 (10.6 %). Hydronephrosis/megaureter and late onset of oligohydramnios were prognostic factors for fetal survival, whereas renal agenesis and associated anomalies had a negative impact on pregnancy outcome. Prenatal interventions did not improve prognosis., Conclusions: Pregnancies complicated by renal oligohydramnios still have a poor outcome. Careful weighing of prognostic factors is necessary to decide about further therapeutic measures.
Published: 2015
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47. Infantile fibrosarcoma - an important differential diagnosis of congenital vascular tumors.

Author: Kerl K, Nowacki M, Leuschner I, Masjosthusmann K, and Frühwald MC
Subjects: Antineoplastic Combined Chemotherapy Protocols therapeutic use, Diagnosis, Differential, Female, Fibrosarcoma drug therapy, Hemangioma drug therapy, Humans, Infant, Newborn, Prognosis, Fibrosarcoma congenital, Fibrosarcoma diagnosis, Hemangioma congenital, Hemangioma diagnosis
Abstract: We present the case of a female newborn with life-threatening bleeding of a ruptured infantile fibrosarcoma (IFS) and consecutive multiorgan dysfunction syndrome shortly after birth. After stabilization, the tumor could be treated without amputation due to surgery, laser therapy, and chemotherapy. The patient is free of tumor and doing functionally well after 4 years of follow-up.
Published: 2012
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48. Circadian variation on oxygen consumption in preterm infants.

Author: Bauer J, Janecke A, Gerss J, Masjosthusmann K, Werner C, and Hoffmann G
Subjects: Calorimetry, Indirect, Humans, Infant, Newborn, Circadian Rhythm, Infant, Premature metabolism, Oxygen Consumption
Abstract: Objective: We investigated the diurnal variation in oxygen consumption to determine the optimal time periods of calorimetry in preterm infants., Methods: Oxygen consumption (VO(2)) was measured continuously for 24 h using indirect calorimetry. Twenty-two premature infants with gestational age of 27-31 (31+/-1.7) weeks were enrolled in the study. Heart rate, respiratory rate, oxygen saturation, skin and rectal temperature and physical activity were monitored continuously., Results: The averaged values of VO(2) showed a significant pattern (P<0.0001) of circadian rhythm with a peak in the afternoon and a nadir during the night with significantly differences between the mean VO(2) values. A circadian variation of VO(2) was found in more than 80% of preterm infants and was unrelated to gestational age, physical activity or environmental stress. The infants spent 90.4% of their time sleeping., Conclusions: These findings indicate the possible existence of an endogenous circadian rhythm of VO(2) in preterm infants beginning shortly after birth. Day-night variations of VO(2) should be considered when VO(2) values are extrapolated from short measurement periods to prevent overestimation of values.
Published: 2009
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49. Concomitant Ewing sarcoma and acute lymphoblastic leukemia in a 5-year-old girl.

Author: Masjosthusmann K, Bielack SS, Köhler G, Florax A, Schiborr M, Bruch J, Reinhardt D, Kuhn N, Paulussen M, and Jürgens H
Subjects: Child, Preschool, Combined Modality Therapy, Core Binding Factor Alpha 2 Subunit, Disease-Free Survival, Female, Humans, Oncogene Proteins, Fusion, Polymerase Chain Reaction, Precursor Cell Lymphoblastic Leukemia-Lymphoma diagnosis, Precursor Cell Lymphoblastic Leukemia-Lymphoma therapy, Proto-Oncogene Protein c-fli-1, RNA-Binding Protein EWS, Sarcoma, Ewing diagnosis, Sarcoma, Ewing therapy, Translocation, Genetic, Precursor Cell Lymphoblastic Leukemia-Lymphoma complications, Sarcoma, Ewing complications
Abstract: Malignancies from the Ewing family of tumors and acute lymphoblastic leukemia (ALL) are not known to be associated with each other. A 5-year-old girl was incidentally found to suffer from acute lymphoblastic leukemia during bone marrow staging for Ewing sarcoma of the radius. The simultaneous presence of two distinct neoplasms was confirmed by RT-PCR, with EWS/FLI1 type 1 rearrangement in the bone tumor and TEL/AML1 rearrangement in the marrow. She was treated with chemotherapy, radiotherapy, and surgery and was in remission of both diseases 31 months after diagnosis., ((c) 2005 Wiley-Liss, Inc.)
Published: 2005
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