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5. Impact of Treosulfan Exposure on Early and Long-Term Clinical Outcomes in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation Recipients: A Prospective Multicenter Study

Author

van der Stoep, M. Y. E. C., Bertaina, A., Moes, D. J. A. R., Algeri, M., Bredius, R. G. M., Smiers, F. J. W., Berghuis, D., Buddingh, E. P., Mohseny, A. B., Guchelaar, H. -J., Locatelli, Franco, Zwaveling, J., Lankester, A. C., Locatelli F. (ORCID:0000-0002-7976-3654), van der Stoep, M. Y. E. C., Bertaina, A., Moes, D. J. A. R., Algeri, M., Bredius, R. G. M., Smiers, F. J. W., Berghuis, D., Buddingh, E. P., Mohseny, A. B., Guchelaar, H. -J., Locatelli, Franco, Zwaveling, J., Lankester, A. C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Treosulfan-based conditioning has gained popularity in pediatric allogeneic hematopoietic stem cell transplantation (HSCT) because of its presumed favorable efficacy and toxicity profile. Treosulfan is used in standardized dosing regimens based on body surface area. The relationships between systemic treosulfan exposure and early and long-term clinical outcomes in pediatric patients undergoing allogeneic HSCT for nonmalignant diseases remain unclear. In this a multicenter, prospective observational study, we assessed the association between treosulfan exposure and early and, in particular, long-term clinical outcomes. Our study cohort comprised 110 pediatric patients with nonmalignant diseases who underwent HSCT between 2011 and 2019 in Leiden, The Netherlands and Rome, Italy. Blood samples were collected, and treosulfan area under the receiver operating characteristic curve (AUC0-∞) was estimated as a measure of exposure. Cox proportional hazard survival analyses were performed to assess the relationships between treosulfan exposure and overall survival (OS) and event-free survival (EFS). The predictive value of systemic treosulfan exposure for the occurrence of toxicity within 28 days was evaluated using a multivariable logistic regression analysis. In the overall cohort, OS and EFS at 2 years were 89.0% and 75.3%, respectively, with an excellent OS of 97% in children age <2 years. The occurrence of grade II-IV acute graft-versus-host disease, the level of 1-year whole blood chimerism, and 2-year OS and EFS were not correlated with treosulfan exposure. The occurrence of skin toxicity (odds ratio [OR], 3.97; 95% confidence interval [CI], 1.26-13.68; P = .02) and all-grade mucositis (OR, 4.43; 95% CI, 1.43-15.50; P = .02), but not grade ≥2 mucositis (OR, 1.51; 95% CI, 0.52 to 4.58; P = .46) was related to high treosulfan exposure (>1750 mg*h/L). Our study demonstrates that standardized treosulfan-based conditioning results in a favorable OS and EFS in infants

Published
2022

6. Busulfan–fludarabine- or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major

Author

Luftinger, R., Zubarovskaya, N., Galimard, J. -E., Cseh, A., Salzer, E., Locatelli, Franco, Algeri, M., Yesilipek, A., de la Fuente, J., Isgro, A., Alseraihy, A., Angelucci, E., Smiers, F. J., La La Nasa, G., Zecca, M., Fisgin, T., Unal, E., Kleinschmidt, K., Peters, C., Lankester, A., Corbacioglu, S., Locatelli F. (ORCID:0000-0002-7976-3654), Luftinger, R., Zubarovskaya, N., Galimard, J. -E., Cseh, A., Salzer, E., Locatelli, Franco, Algeri, M., Yesilipek, A., de la Fuente, J., Isgro, A., Alseraihy, A., Angelucci, E., Smiers, F. J., La La Nasa, G., Zecca, M., Fisgin, T., Unal, E., Kleinschmidt, K., Peters, C., Lankester, A., Corbacioglu, S., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Significant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients’ life expectancy. However, transfusion-associated iron overload remains a significant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of the best available donor, an optimized conditioning regimen is crucial to maximize outcomes for patients with TDT undergoing HSCT. The aim of this retrospective analysis was to investigate the role of busulfan–fludarabine-based and treosulfan–fludarabine-based conditioning in TDT patients undergoing HSCT. We included 772 patients registered in the European Society for Blood and Marrow Transplantation (EBMT) database who underwent first HSCT between 2010 and 2018. Four hundred ten patients received busulfan–fludarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan–fludarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confidence interval: 89.3–95.1%) after busulfan–fludarabine-based conditioning and 94.7% (95% confidence interval: 91.7–96.6%) after treosulfan–fludarabine-based conditioning. There was a very low incidence of second HSCT overall. The main causes of death were infections, graft-versus-host disease, and rejection. In conclusion, use of busulfan or treosulfan as the backbone of myeloablative conditioning for patients with TDT undergoing HSCT resulted in comparably high cure rates. Long-term follow-up studies are warranted to address the important issues of organ toxicities and gonadal function.

Published
2022

8. BMI is an important determinant of VWF and FVIII levels and bleeding phenotype in patients with von Willebrand disease

Author

Atiq, Ferdows, Fijnvandraat, Karin, van Galen, Karin P. M., Laros-van Gorkom, Britta A. P., Meijer, Karina, de Meris, Joke, Coppens, Michiel, Mauser-Bunschoten, Eveline P., Cnossen, Marjon H., van der Bom, Johanna G., Eikenboom, Jeroen, Leebeek, Frank W. G., Coppens, M., Kors, A., Zweegman, S., de Meris, J., Goverde, G. J., Jonkers, M. H., Dors, N., Nijziel, M. R., Nieuwenhuizen, L., Meijer, K., Tamminga, R. Y. J., van der LindenHagaZiekenhuis, P. W., Ypma, P. F., Eikenboom, H. C. J., van der Bom, J. G., Smiers, F. J. W., Granzen, B., Hamulyák, K., Brons, P., Laros‐van Gorkom, B. A. P., Leebeek, F. W. G., Cnossen, M. H., Atiq, F., Mauser-Bunschoten, E. P., van Galen, K. P. M., Hematology, Pediatrics, Paediatric Haematology, ACS - Pulmonary hypertension & thrombosis, Vascular Medicine, ARD - Amsterdam Reproduction and Development, VU University medical center, Kindergeneeskunde, MUMC+: MA Medische Staf Kindergeneeskunde (9), RS: FHML non-thematic output, and Real World Studies in PharmacoEpidemiology, -Genetics, -Economics and -Therapy (PEGET)

Subjects
Male, medicine.medical_specialty, Letter, Vascular damage Radboud Institute for Health Sciences [Radboudumc 16], E–Only Articles, Hemorrhage, von Willebrand Disease, Type 2, von Willebrand Disease, Type 1, Body Mass Index, Text mining, Internal medicine, von Willebrand Factor, Correspondence, Von Willebrand disease, Medicine, Humans, In patient, Hematology, Factor VIII, business.industry, Middle Aged, Blood coagulation factors, medicine.disease, Phenotype, Immunology, Female, business, Body mass index
Abstract

Contains fulltext : 208376.pdf (Publisher’s version ) (Open Access)

Published
2019

9. Inhibitor development and mortality in non-severe hemophilia A

Author

Eckhardt, C. L., Loomans, J. I., van Velzen, A. S., Peters, M., Mauser-Bunschoten, E. P., Schwaab, R., Mazzucconi, M. G., Tagliaferri, A., Siegmund, B., Reitter-Pfoertner, S. E., van der Bom, J. G., Fijnvandraat, K., Kamphuisen, P. W., Peerlinck, K., Oldenburg, J., Santagostino, E., Astermark, J., Eckhardt, C. L, van Velzen, A. S, Streefkerk, N., Loomans, J. L., van Eijkelenburg, A., Jansen, A. J., Kruijt, C. C., van Tienoven, B., van Baar, A. C. G., Corten, I. W., Meijer, K., Nijziel, M. R., Dors, N., Hamulyak, K., Beckers, E., Brons, P. P., Laros-van Gorkom, B. A. P., van Heerde, W. L., Leebeek, F., Kruip, M., Cnossen, M. H., Mauser-Bunschoten, E., Fischer, K., Smiers, F. J., Hermans, C., Klamroth, R., Escuriola-Ettingshausen, C., Königs, C., Petrini, P., Holmström, M., Mäkipernaa, A., Male, C., Pabinger, I., Keenan, R. D., Liesner, R., Khair, K., Yee, T. T., Hart, D. P., Rangarajan, S., Mitchell, M., Thompson, G., Haya, S., Moret, A., Cid, A. R., Jimenez-Yuste, V., Mancuso, M. E., Mazzuconni, M. G., Santoro, C., Morfini, M., Castaman, G., Schinco, P., Rivolta, G. F., Platokouki, H., and McRae, S.

Published
2015
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11. CLEC4M and STXBP5 gene variations contribute to von Willebrand factor level variation in von Willebrand disease

Author

Sanders, Y. V., van der Bom, J. G., Isaacs, A., Cnossen, M. H., de Maat, M. P. M., Laros-van Gorkom, B. A. P., Fijnvandraat, K., Meijer, K., van Duijn, C. M., Mauser-Bunschoten, E. P., Eikenboom, J., Leebeek, F. W. G., Coppens, M., Kors, A., de Meris, J., Nijziel, M. R., Tamminga, R. Y. J., Ypma, P. F., Smiers, F. J. W., Granzen, B., Hamulyák, K., and Brons, P.

Published
2015
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14. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party

Author

Prata, P.H., Eikema, D.J., Afansyev, B., Bosman, P., Smiers, F., Diez-Martin, J.L., Arrais-Rodrigues, C., Koc, Y., Poir?, X., Sirvent, A., Kr?ger, N., Porta, F., Holter, W., Bloor, A., Jubert, C., Ganser, A., Tanase, A., M?nard, A.L., Pioltelli, P., P?rez-Sim?n, J.A., Ho, A., Aljurf, M., Russell, N., Labussiere-Wallet, H., Kerre, T., Rocha, V., Soci?, G., Risitano, A., Dufour, C., Latour, R.P. de, SAA WP EBMT, Prata, P. H., Eikema, D. -J., Afansyev, B., Bosman, P., Smiers, F., Diez-Martin, J. L., Arrais-Rodrigues, C., Koc, Y., Poire, X., Sirvent, A., Kroger, N., Porta, F., Holter, W., Bloor, A., Jubert, C., Ganser, A., Tanase, A., Menard, A. -L., Pioltelli, P., Perez-Simon, J. A., Ho, A., Aljurf, M., Russell, N., Labussiere-Wallet, H., Kerre, T., Rocha, V., Socie, G., Risitano, A., Dufour, C., and Peffault de Latour, R.

Subjects
medicine.medical_specialty, Transplantation Conditioning, Cyclophosphamide, medicine.medical_treatment, Eltrombopag, Graft vs Host Disease, Gastroenterology, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Internal medicine, Medicine, Humans, Cumulative incidence, Prospective Studies, Aplastic anemia, Prospective cohort study, Transplantation, Univariate analysis, Neutrophil Engraftment, business.industry, Hematopoietic Stem Cell Transplantation, Anemia, Aplastic, Immunosuppression, Hematology, medicine.disease, Europe, chemistry, 030220 oncology & carcinogenesis, Transplantation, Haploidentical, business, 030215 immunology, medicine.drug
Abstract

In Press., In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI95%: 51–83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II–III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0–20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64–93), and 2-year graft-versus-host disease-free survival was 63% (46–81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81–100) versus 64% (41–87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.

Published
2019

16. Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT)

Author

Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., Locatelli F. (ORCID:0000-0002-7976-3654), Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative procedure in patients with Shwachman–Diamond syndrome (SDS) with bone marrow abnormalities. The results of 74 patients with SDS (6 acute myeloid leukemia, 7 myelodysplastic syndrome, and 61 bone marrow failure) treated with HSCT between 1988 and 2016 are reported. The donor source was: 24% sibling, 8% parent, and 68% unrelated donor. The stem cell source was: 70% bone marrow, 19% peripheral blood stem cells, and 11% cord blood. The conditioning regimen was myeloablative in 54% and reduced intensity in 46%. Neutrophil engraftment was achieved in 84% of patients after a median time of 17.5 days. Graft failure occurred in 15% of HSCTs. Grades I–IV acute and chronic GVHD were observed in 55% and 20% of patients, respectively. After a median follow-up of 7.3 years (95% CI 4.8–10.2), 28 patients died for progression/relapse (7) or toxicity (21). The 5-year overall survival and nonrelapse mortality were 63.3% (95% CI 50.8–73.4) and 19.8% (95% CI 10.8–30.8), respectively. In conclusion, this is the largest series so far reported and confirms that HSCT is a suitable option for patients with SDS. Further efforts are needed to lower transplant-related toxicity and reduce graft failure.

Published
2020

17. Correction: Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT) (Bone Marrow Transplantation, (2020), 55, 9, (1796-1809), 10.1038/s41409-020-0863-z)

Author

Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., Locatelli F. (ORCID:0000-0002-7976-3654), Cesaro, S., Pillon, M., Sauer, M., Smiers, F., Faraci, M., de Heredia, C. D., Wynn, R., Greil, J., Locatelli, Franco, Veys, P., Uyttebroeck, A., Ljungman, P., Chevalier, P., Ansari, M., Badell, I., Gungor, T., Salim, R., Tischer, J., Tecchio, C., Russell, N., Chybicka, A., Styczynski, J., Krivan, G., Smith, O., Stein, J., Afanasyev, B., Pochon, C., Menconi, M. C., Bosman, P., Mauro, M., Tridello, G., de Latour, R. P., Dufour, C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Published
2020

25. von Willebrand factor propeptide and the phenotypic classification of von Willebrand disease

Author

Sanders, Yvonne V., Groeneveld, Dafna, Meijer, Karina, Fijnvandraat, Karin, Cnossen, M. H., Van Der Bom, J. G., Coppens, M., De Meris, Joke, Laros-Van Gorkom, B. A.P., Mauser-Bunschoten, Eveline P., Leebeek, F. W.G., Eikenboom, Jeroen, Fijnvandraat, K., Kors, A., Zweegman, S., De Meris, J., Goverde, G. J., Jonkers, M. H., Dors, N., Nijziel, M. R., Meijer, K., Tamminga, R. Y.J., Van Der Linden, P. W., Ypma, P. F., Eikenboom, J., Smiers, F. J.W., Granzen, B., Hamulyák, K., Brons, P., Sanders, Y. V., RS: GROW - Developmental Biology, RS: GROW - R4 - Reproductive and Perinatal Medicine, Hematology, Pediatrics, CCA - Disease profiling, CCA - Innovative therapy, CCA - Quality of life, Anatomy and neurosciences, NCA - Neuroinflamation, Vascular Ageing Programme (VAP), Real World Studies in PharmacoEpidemiology, -Genetics, -Economics and -Therapy (PEGET), ACS - Amsterdam Cardiovascular Sciences, AII - Amsterdam institute for Infection and Immunity, Paediatric Infectious Diseases / Rheumatology / Immunology, Vascular Medicine, and Other departments

Subjects
Male, VONWILLEBRAND-FACTOR, CLEARANCE, Vascular damage Radboud Institute for Health Sciences [Radboudumc 16], Biochemistry, hemic and lymphatic diseases, Missense mutation, ASSAY, ADULT PATIENTS, Child, Netherlands, Aged, 80 and over, biology, FACTOR-VIII, Hematology, Middle Aged, Prognosis, QUANTITATIVE-ANALYSIS, Null allele, Pathophysiology, von Willebrand Diseases, Phenotype, Child, Preschool, cardiovascular system, VWF PROPEPTIDE, Female, circulatory and respiratory physiology, Adult, medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, Adolescent, Immunology, Hemorrhage, Rare cancers Radboud Institute for Molecular Life Sciences [Radboudumc 9], DIAGNOSIS, Young Adult, Von Willebrand factor, Antigen, Internal medicine, von Willebrand Factor, Von Willebrand disease, medicine, Humans, Clinical significance, Protein Precursors, Protein precursor, Aged, MULTIMERIZATION, business.industry, Infant, Cell Biology, medicine.disease, Cross-Sectional Studies, Endocrinology, Mutation, biology.protein, business, FACTOR SURVIVAL, Follow-Up Studies
Abstract

Item does not contain fulltext The ratios between von Willebrand factor propeptide (VWFpp) or factor VIII activity ( FVIII: C) and VWF antigen (VWF:Ag) reflect synthesis, secretion, and clearance of VWF. We aimed to define the pathophysiology of 658 patients with type 1, 2, or 3 von Willebrand disease (VWD) with VWF levels

Published
2015

28. STEM CELL TRANSPLANTATION IN PYRUVATE KINASE DEFICIENCY

Author

Straaten, S. van, Bierings, M., Bianchi, P., Akiyoshi, K., Kanno, H., Serra, I.B., Chen, J., Huang, X., Beers, E. van, Ekwattanakit, S., Gungor, T., Kors, W.A., Smiers, F., Raymakers, R., Yanez, L., Sevilla, J., Solinge, W. van, Segovia, J.C., and Wijk, R. van

Published
2017

29. High interpatient variability of treosulfan exposure is associated with early toxicity in paediatric HSCT: a prospective multicentre study

Author

van der Stoep, M. Y. E. C., Bertaina, A., ten Brink, M. H., Bredius, R. G., Smiers, F. J., Wanders, D. C. M., Moes, D. J. A. R., Locatelli, Franco, Guchelaar, H. -J., Zwaveling, J., Lankester, A. C., Locatelli F. (ORCID:0000-0002-7976-3654), van der Stoep, M. Y. E. C., Bertaina, A., ten Brink, M. H., Bredius, R. G., Smiers, F. J., Wanders, D. C. M., Moes, D. J. A. R., Locatelli, Franco, Guchelaar, H. -J., Zwaveling, J., Lankester, A. C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Treosulfan-based conditioning is increasingly employed in paediatric haematopoietic stem cell transplantation (HSCT). Data on treosulfan pharmacokinetics in children are scarce, and the relationship between treosulfan exposure, toxicity and clinical outcome is unresolved. In this multicentre prospective observational study, we studied treosulfan pharmacokinetics and the drug's relationship with regimen-related toxicity and early clinical outcome in 77 paediatric patients. Treosulfan dose was 30 g/m2, administered over 3 consecutive days in infants <1 year old (n = 12) and 42 g/m2 in children ≥1 year old (n = 65). Mean day 1 treosulfan exposure was 1744 ± 795 mg*h/l (10 g/m2) and 1561 ± 511 mg*h/l (14 g/m2), with an inter-individual variability of 56 and 33% in the respective groups. High treosulfan exposure (>1650 mg*h/l) was associated with an increased risk of mucosal [Odds ratio (OR) 4·40; 95% confidence interval (CI) 1·19–16·28, P = 0·026] and skin toxicity (OR 4·51; 95% CI 1·07–18·93, P = 0·040). No correlation was found between treosulfan exposure and the early clinical outcome parameters: engraftment, acute graft-versus-host disease and donor chimerism. Our study provides the first evidence in a large cohort of paediatric patients of high variability in treosulfan pharmacokinetics and an association between treosulfan exposure and early toxicity. Ongoing studies will reveal whether treosulfan exposure is related to long-term disease-specific outcome and late treatment-related toxicity.

Published
2017

31. Von Willebrand disease and aging: An evolving phenotype

Author

Sanders, Y. V., Giezenaar, M. A., Laros-van Gorkom, B. A.P., Meijer, K., van der Bom, J. G., Cnossen, M. H., Nijziel, M. R., Ypma, P. F., Fijnvandraat, K., Eikenboom, J., Mauser-Bunschoten, E. P., Leebeek, F. W.G., Middeldorp, S., Kors, A., Zweegman, S., de Meris, J., Jonkers, M. H., Dors, N., Tamminga, R. Y.J., van der Linden, P. W., Eikenboom, H. C.J., Smiers, F. J.W., Granzen, B., Hamulyak, K., Brons, P., Hematology, Epidemiology, Pediatrics, Nutrition and Movement Sciences, Kindergeneeskunde, RS: GROW - Developmental Biology, RS: GROW - R4 - Reproductive and Perinatal Medicine, Vascular Ageing Programme (VAP), ACS - Amsterdam Cardiovascular Sciences, AII - Amsterdam institute for Infection and Immunity, Paediatric Infectious Diseases / Rheumatology / Immunology, Vascular Medicine, Other departments, NCA - Neuroinflamation, CCA - Disease profiling, and CCA - Innovative therapy

Subjects
Male, Pediatrics, Cross-sectional study, Vascular damage Radboud Institute for Health Sciences [Radboudumc 16], von Willebrand factor, Gastroenterology, von Willebrand Diseases: therapy, Quality of life, QUALITY-OF-LIFE, hemic and lymphatic diseases, UPPER GASTROINTESTINAL HEMORRHAGE, 80 and over, ADULT PATIENTS, Young adult, POPULATION, Netherlands, Aged, 80 and over, education.field_of_study, HEMOPHILIA, biology, Age Factors, Hematology, Middle Aged, Phenotype, humanities, Pathophysiology, INHERITED BLEEDING DISORDERS, Hospitalization, von Willebrand Diseases, factor VIII, Female, ELDERLY PERSONS, von Willebrand Diseases: physiopathology, hemorrhage, von Willebrand disease, circulatory and respiratory physiology, Adult, medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, Adolescent, Population, CO-MORBIDITY, Young Adult, Von Willebrand factor, Internal medicine, MANAGEMENT, medicine, Von Willebrand disease, Humans, education, Aged, business.industry, aging, von Willebrand Factor: metabolism, medicine.disease, Cross-Sectional Studies, biology.protein, MODERATE, business
Abstract

Item does not contain fulltext BACKGROUND: Because the number of elderly von Willebrand disease (VWD) patients is increasing, the pathophysiology of aging in VWD has become increasingly relevant. OBJECTIVES: To assess age-related changes in von Willebrand factor (VWF) and factor VIII (FVIII) levels and to compare age-related differences in bleeding phenotype between elderly VWD patients and those < 65 years. We also studied co-morbidity in elderly patients. PATIENTS/METHODS: We included VWD patients with VWF levels

Published
2014

32. Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

Author

Angelucci, E., Matthes-Martin, S., Baronciani, D., Bernaudin, F., Bonanomi, S., Cappellini, M.D., Dalle, J.H., Bartolomeo, P. di, Heredia, C.D. de, Dickerhoff, R., Giardini, C., Gluckman, E., Hussein, A.A., Kamani, N., Minkov, M., Locatelli, F., Rocha, V., Sedlacek, P., Smiers, F., Thuret, I., Yaniv, I., Cavazzana, M., Peters, C., and EBMT Inborn Error EBMT Paediat Wor

Subjects
medicine.medical_specialty, Anemia, Thalassemia, medicine.medical_treatment, Cell, Anemia, Sickle Cell, Disease, Hematopoietic stem cell transplantation, Guideline Article, medicine, Humans, Child, Intensive care medicine, business.industry, Marrow transplantation, beta-Thalassemia, Hematopoietic Stem Cell Transplantation, Hematology, medicine.disease, Surgery, surgical procedures, operative, medicine.anatomical_structure, N/A, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Savior sibling, business
Abstract

Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.

Published
2014

34. Inhibitor eradication therapy in non-severe hemophilia A

Author

Velzen, A.S. van, Eckhardt, C.L., Peters, M., Hart, P., Rangarajan, S., Hay, C.R.M., Mancuso, M.E., Smiers, F., Liesner, R., Petrini, P., Yuste, V.J., Bom, J.G. van der, Fijnvandraat, K.J., Yee, T., and INSIGHT Consortium

Published
2013

39. Veneuze trombo-embolie bij kinderen: diagnostiek, preventie en behandeling.

Author

Ommen, C., Smiers, F., and Appel, I.

Abstract

Copyright of Tijdschrift Voor Kindergeneeskunde is the property of Springer Nature and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

Published
2007
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40. Paediatric allogeneic bone marrow transplantation for homozygous B-thalassaemia, the Dutch experience.

Author

Ball, L M, Lankester, A C, Giordano, P C, van Weel, M H, Harteveld, C L, Bredius, R G M, Smiers, F J, Egeler, R M, and Vossen, J M J J

Subjects
BONE marrow transplantation, PEDIATRICS, TRANSPLANTATION immunology, GRAFT versus host disease, GLOBIN
Abstract

Summary:We reviewed the results of the Dutch paediatric bone marrow transplant (BMT) program for children receiving HLA-identical BMT for B-thalassaemia major over an 18-year period. In all, 19 patients underwent a total of 21 transplants in our treatment centre between July 1984 and February 2002. Eight females (age 0.3-12 years; median 5 years) and 11 males (age 0.8-18 years; median 6 years) were included. Information, prospectively collected, included molecular defects, donor genotype, B/a-globin expression rates, serum ferritin levels, hepato-splenomegaly, chelation history, virology screening, liver pathology together with post-transplant outcome inclusive of leucocyte chimerism. In total, 11 patients received standard busulphan/cyclophosphamide (Bu/Cy) conditioning, with or without ATG. Stable engraftment was seen in 5/11 with late rejection occurring in six patients. Of these, two children underwent a second successful SCT. For this group, overall event-free survival (EFS) and disease-free survival (DFS) were 90 (10/11) and 64% (7/11), respectively. The probability of rejection was 55%. Subsequent addition of melphalan to the conditioning regimen resulted in long-term stable engraftment in all patients with an EFS/DFS for this group of 90% (9/10). Treatment-related mortality, irrespective of conditioning, was low at 5% (1/19 patients). Veno-occlusive disease (VOD) occurred in 19% (4/21 transplants) and acute GvHD in 19% (4/21 transplants). Post-BMT B/a synthetic ratio measurement monitored donor erythroid engraftment and predicted rejection with a return to transfusion dependency. Maintained full donor chimerism is indicative of stable engraftment both for leucocyte and erythroid lineages, whereas mixed donor chimerism is not. Our results emphasise the importance of the conditioning regimen and post-transplant chimerism surveillance predictive of rejection or long-term stable engraftment.Bone Marrow Transplantation (2003) 31, 1081-1087. doi:10.1038/sj... [ABSTRACT FROM AUTHOR]

Published
2003
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41. Hoping for a normal life: Decision-making on hematopoietic stem cell transplantation by patients with a hemoglobinopathy and their caregivers.

Author

Mekelenkamp H, de Vries M, Saalmink I, Nur E, Kerkhoffs JL, Heijboer H, Cnossen M, Lankester A, and Smiers F

Subjects
Child, Young Adult, Humans, Caregivers, Patients, Anemia, Sickle Cell therapy, Hematopoietic Stem Cell Transplantation
Abstract

Background: To provide insight into the perspectives of children and young adults with transfusion-dependent thalassemia and sickle cell disease and their caregivers regarding the decision for hematopoietic stem cell transplantation (HSCT)., Procedure: A qualitative longitudinal multicenter study. Data collection consisted of 40 audio-recorded conversations between physicians and families and 77 interviews with patients and/or caregivers related to 27 unique cases, collected at different time points throughout the decision-making process., Results: Conversations and interviews revealed "hoping for a normal life" as an overarching theme, consisting of four main topics: (i) "Building a frame of reference" refers to a process where patients or families try to obtain comprehensive information on HSCT and translate this to their situation to decide. (ii) "Balancing between loss and benefit" reports the process of considering the advantages and disadvantages of continuing with supportive care to treat their disease versus choosing HSCT. (iii) "Experiencing the impact of HSCT" describes the impactfull experience of the HSCT period by those who chose HSCT. (iv) "Balancing again" refers to reflecting on the decision made., Conclusions: The hope for a normal life guided the decision-making process, described as a constant balance between the impact of the disease and HSCT. A structured approach to explore patients' and caregivers' perspectives on HSCT decision-making is needed, where specifically discussing the impact of the disease and hope for a normal life need to be integrated in the process., (© 2023 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)

Published
2024
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42. Optimizing diagnostic methods and stem cell transplantation outcomes in pediatric bone marrow failure: a 50-year single center experience.

Author

Vissers L, van der Burg M, Lankester A, Smiers F, and Mohseny A

Subjects
Child, Humans, Congenital Bone Marrow Failure Syndromes etiology, Bone Marrow Failure Disorders diagnosis, Bone Marrow Failure Disorders etiology, Anemia, Aplastic diagnosis, Anemia, Aplastic therapy, Hematopoietic Stem Cell Transplantation adverse effects, Hematopoietic Stem Cell Transplantation methods
Abstract

Peripheral blood cytopenia, a frequent presenting symptom in pediatric patients, can be caused by bone marrow failure (BMF). Timely identification of patients with non-reversible BMF is of crucial importance to reduce the risks of invasive infections and bleeding complications. Most pediatric patients with severe persistent cytopenia, independent of the underlying cause, are offered allogeneic hematopoietic stem cell transplantation (HSCT) as curative therapy. Here we report on our management guidelines and HSCT outcomes of pediatric BMF patients to pinpoint improvements and future challenges. We formulated recommendations based on this 50 years' experience, which were implemented at our center in 2017. By analysis of the HSCT cohort of 2017-2023, the 5-year outcome data is presented and compared to historical outcome data. In addition, outcomes of patients transplanted for identified inherited bone marrow failure syndromes (IBMFS) are compared to severe aplastic anemia (SAA) outcomes to underline the often multiorgan disease in IBMFS with implications for long-term survival. Survival of pediatric patients with irreversible BMF has improved tremendously. SAA patients transplanted after 2017 had a superior 5-year overall (OS) and event-free survival (EFS) of 97% and 85% compared to 68% and 59% in the cohort transplanted before 2017 (p = 0.0011 and p = 0.017). A similar trend was seen for BMF, with an OS and EFS of 89% for those transplanted after 2017 compared to 62% and 59% (p > 0.05). This improvement is mainly related to better survival in the first months after HSCT. The long-term survival after HSCT is lower in IBMFS patients as compared to SAA patients due to secondary malignancies and multiorgan toxicity.   Conclusion: Unbiased protocolized in-depth diagnostic strategies are crucial to increase the frequency of identifiable causes within the heterogeneous group of pediatric BMF. A comprehensive approach to identify the cause of BMF can prevent treatment delay and be useful to tailor treatment and follow-up protocols. What is Known: • Irreversible BMF in pediatric patients can be caused by a wide spectrum of underlying diseases including (pre)malignant disease, IBMFS and AA. Identifying the exact underlying cause of BMF is crucial for tailored therapy, however often challenging and time-consuming. • Frontline allogeneic HSCT is offered to most pediatric patients with severe BMF as curative treatment. What is New: • Protocolized unbiased diagnostics, short time to treatment (< 3 months) and maximal supportive care until curative treatment can prevent complications with a negative effect on survival such as infection and bleeding. • Personalized follow-up protocols for IBMFS patients are essential to prevent a second decline in survival due to long-term treatment toxicity and extra-hematological disease complications., (© 2023. The Author(s).)

Published
2023
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44. Decision making for hematopoietic stem cell transplantation in pediatric, adolescent, and young adult patients with a hemoglobinopathy-Shared or not?

Author

Mekelenkamp H, Smiers F, Camp N, Stubenrouch F, Lankester A, and de Vries M

Subjects
Adolescent, Child, Communication, Humans, Physician-Patient Relations, Young Adult, Decision Making, Shared, Hematopoietic Stem Cell Transplantation, Hemoglobinopathies psychology, Hemoglobinopathies therapy, Patient Participation
Abstract

Background: Hematopoietic stem cell transplantation (HSCT) offers an established curative option for sickle cell disease (SCD) and thalassemia patients but is associated with significant risks. Decision making is a complex process and shared decision making (SDM) could be a fitting approach in case of such preference-sensitive decisions. This study investigated what level of SDM is used in conversations with hemoglobinopathy patients and/or their caregivers considering HSCT as a curative treatment option., Methods: Longitudinal, descriptive study using the Observing-Patient-Involvement-in-Decision-Making scale (OPTION 5 ) scale to determine the level of SDM in conversations with 26 hemoglobinopathy patients and/or their caregivers., Results: The total mean OPTION 5 score was 43%, which is a moderate SDM approach. There was no difference between conversations with thalassemia patients and SCD patients. Conversations needing an interpreter scored worse than nontranslated conversations. The best scoring OPTION 5 item was item 3: "informing about the various treatment options" (mean score 2.3 on scale 0-4). For OPTION 5 item 4: "eliciting patients' preferences" a more skilled effort was measured for SCD patients compared to thalassemia patients., Conclusions: The mean OPTION 5 score of "moderate" was achieved mainly by giving information on available options, which is primarily a one-way communication. The SDM process can be improved by actively inviting patients to deliberate about options and including their elicited preferences in decision making., (© 2021 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)

Published
2021
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45. How to Facilitate Decision-Making for Hematopoietic Stem Cell Transplantation in Patients With Hemoglobinopathies. The Perspectives of Healthcare Professionals.

Author

Mekelenkamp H, van Zanten H, de Vries M, Lankester A, and Smiers F

Abstract

Hematopoietic stem cell transplantation decision-making for hemoglobinopathy patients is a complex process, and it remains difficult for health care professionals to decide whether and when a hematopoietic stem cell transplantation should be offered. Gaining insight into health care professionals' considerations is required to understand and optimize this decision-making process. A qualitative interview study using semi-structured interviews with eighteen health care professionals. Data were thematically analyzed. Two main themes emerged from the data: (1) Experiencing the influence of a frame of reference and (2) Feeling responsible for a guided decision-making . The frame of reference, meaning the health care professionals' knowledge and experiences regarding hematopoietic stem cell transplantation, influenced the guided decision-making process. Subsequently, three subthemes evolved from the second theme: (a) weighing up disease severity against possible complications, (b) making an effort to inform, and (c) supporting the best fitting decision for the individual patient. The health care professionals' frame of reference determined the hematopoietic stem cell transplantation decision-making process. This demands reflection on the health care professionals' own frame of reference and its influence on decision-making. Furthermore, reflection on the frame of reference is needed by exchange of knowledge and experiences between referring and referred-to healthcare professionals in an open and two-way direction. The transplantation teams have a responsibility of keeping the frame of reference of their referring colleagues up to date and referring health care professionals should share their feelings regarding hematopoietic stem cell transplantation. To guide patients, a shared decision-making approach is supportive, in which eliciting the patients' preferences is highly important. Health care professionals can refine the decision-making process by guiding patients in eliciting their preferences and including these in the decision., 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 Mekelenkamp, Zanten, Vries, Lankester and Smiers.)

Published
2021
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46. Stem Cell Transplantation for Diamond-Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT).

Author

Miano M, Eikema DJ, de la Fuente J, Bosman P, Ghavamzadeh A, Smiers F, Sengeløv H, Yesilipek A, Formankova R, Bader P, Díaz Pérez MÁ, Bertrand Y, Niemeyer C, Diallo S, Ansari M, Bykova TA, Faraci M, Bonanomi S, Gozdzik J, Satti TM, Bodova I, Wölfl M, Rocha VG, Mellgren K, Rascon J, Holter W, Lange A, Meisel R, Beguin Y, Mozo Y, Kriván G, Sirvent A, Bruno B, Dalle JH, Onofrillo D, Giardino S, Risitano AM, de Latour RP, and Dufour C

Subjects
Bone Marrow, Child, Humans, Retrospective Studies, Anemia, Aplastic therapy, Anemia, Diamond-Blackfan therapy, Hematopoietic Stem Cell Transplantation
Abstract

Data on stem cell transplantation (SCT) for Diamond-Blackfan Anemia (DBA) is limited. We studied patients transplanted for DBA and registered in the EBMT database. Between 1985 and 2016, 106 DBA patients (median age, 6.8 years) underwent hematopoietic stem cell transplantation from matched-sibling donors (57%), unrelated donors (36%), or other related donors (7%), using marrow (68%), peripheral blood stem cells (20%), both marrow and peripheral blood stem cells (1%), or cord blood (11%). The cumulative incidence of engraftment was 86% (80% to 93%), and neutrophil recovery and platelet recovery were achieved on day +18 (range, 16 to 20) and +36 (range, 32 to 43), respectively. Three-year overall survival and event-free survival were 84% (77% to 91%) and 81% (74% to 89%), respectively. Older patients were significantly more likely to die (hazard ratio, 1.4; 95% confidence interval, 1.06 to 1.23; P < .001). Outcomes were similar between sibling compared to unrelated-donor transplants. The incidence of acute grades II to IV of graft-versus-host disease (GVHD) was 30% (21% to 39%), and the incidence of extensive chronic GVHD was 15% (7% to 22%). This study shows that SCT may represent an alternative therapeutic option for transfusion-dependent younger patients., (Copyright © 2021. Published by Elsevier Inc.)

Published
2021
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47. The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe.

Author

Gluckman E, Fuente J, Cappelli B, Scigliuolo GM, Volt F, Tozatto-Maio K, Rocha V, Tommaso M, O'Boyle F, Smiers F, Cunha-Riehm CBD, Calore E, Bonanomi S, Graphakos S, Paisiou A, Albert MH, Ruggeri A, Zecca M, Lankester AC, and Corbacioglu S

Subjects
Child, Europe, Humans, Transplantation Conditioning, Anemia, Sickle Cell therapy, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Unrelated Donors
Abstract

We report the results of an analysis of unrelated allogeneic hematopoietic stem cell transplantations (HSCT) in 71 patients with sickle cell disease (SCD) transplanted in EBMT centers between 2005 and 2017. Median age was 9.3 years; graft type was bone marrow in 79% and peripheral blood in 21%. Recipient-donor HLA match at high resolution typing was 10/10 in 31, 9/10 in 20, and 8/10 in 4 patients; the other patients had intermediate resolution typing. The most frequent conditioning regimens were fludarabine-thiotepa-treosulfan (64%) or busulfan-cyclophosphamide (12%). Cumulative incidence of neutrophil engraftment was 92%; platelet engraftment was 90%. Eleven patients (15%) experienced graft failure. Grade II-IV acute graft-vs.-host disease (GvHD) was 23%; 3-year chronic GvHD was 23%. Three-year overall survival (OS) was 88 ± 4%. GRFS was 62 ± 6%. HLA matching was the most significant risk factor for OS: 3-year OS was 96 ± 4% in 10/10 group vs. 75 ± 10% in 9-8/10 (p = 0.042); GRFS was 69 ± 9% vs. 50 ± 12% (p = 0.114), respectively. In conclusion, unrelated donor HSCT is a valid option for SCD patients who lack an HLA-identical sibling donor, preferably in the context of clinical trials. Using a 10/10 HLA-matched unrelated donor yields better survival indicating that HLA matching is an important donor selection factor in this nonmalignant disease.

Published
2020
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48. Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT).

Author

Cesaro S, Pillon M, Sauer M, Smiers F, Faraci M, de Heredia CD, Wynn R, Greil J, Locatelli F, Veys P, Uyttebroeck A, Ljungman P, Chevalier P, Ansari M, Badell I, Güngör T, Salim R, Tischer J, Tecchio C, Russell N, Chybicka A, Styczynski J, Krivan G, Smith O, Stein J, Afanasyev B, Pochon C, Menconi MC, Bosman P, Mauro M, Tridello G, de Latour RP, and Dufour C

Subjects
Bone Marrow, Humans, Retrospective Studies, Shwachman-Diamond Syndrome, Transplantation Conditioning, Treatment Outcome, Anemia, Aplastic therapy, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation
Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative procedure in patients with Shwachman-Diamond syndrome (SDS) with bone marrow abnormalities. The results of 74 patients with SDS (6 acute myeloid leukemia, 7 myelodysplastic syndrome, and 61 bone marrow failure) treated with HSCT between 1988 and 2016 are reported. The donor source was: 24% sibling, 8% parent, and 68% unrelated donor. The stem cell source was: 70% bone marrow, 19% peripheral blood stem cells, and 11% cord blood. The conditioning regimen was myeloablative in 54% and reduced intensity in 46%. Neutrophil engraftment was achieved in 84% of patients after a median time of 17.5 days. Graft failure occurred in 15% of HSCTs. Grades I-IV acute and chronic GVHD were observed in 55% and 20% of patients, respectively. After a median follow-up of 7.3 years (95% CI 4.8-10.2), 28 patients died for progression/relapse (7) or toxicity (21). The 5-year overall survival and nonrelapse mortality were 63.3% (95% CI 50.8-73.4) and 19.8% (95% CI 10.8-30.8), respectively. In conclusion, this is the largest series so far reported and confirms that HSCT is a suitable option for patients with SDS. Further efforts are needed to lower transplant-related toxicity and reduce graft failure.

Published
2020
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49. Management of cerebral azole-resistant Aspergillus fumigatus infection: A role for intraventricular liposomal-amphotericin B.

Author

Schauwvlieghe AFAD, Bredius RGM, Verdijk RM, Smiers FJW, van der Beek MT, Goemans BF, Zwaan CM, Brüggemann RJ, and Rijnders BJA

Subjects
Antifungal Agents pharmacology, Antifungal Agents therapeutic use, Azoles pharmacology, Azoles therapeutic use, Drug Resistance, Fungal, Humans, Amphotericin B pharmacology, Amphotericin B therapeutic use, Aspergillus fumigatus
Abstract

Objectives: In the pre-azole era, central nervous system (CNS) infections with Aspergillus had a dismal outcome. Survival improved with voriconazole but CNS infections caused by azole-resistant Aspergillus fumigatus preclude its use. Intravenous liposomal-amphotericin B (L-AmB) is the preferred treatment option for azole-resistant CNS infections but has suboptimal brain concentrations., Methods: We describe three patients with biopsy-proven CNS aspergillosis where intraventricular L-AmB was added to systemic therapy. Two patients with azole-resistant aspergillosis and one patient with azole-susceptible CNS aspergillosis were treated with intraventricular L-AmB at a dose of 1mg weekly., Results: We describe three patients successfully treated with a combination of intravenous and intraventricular L-AmB. All three patients survived but one patient developed serious headaches, most likely not related to this treatment., Conclusions: Intraventricular L-AmB may have a role in the treatment of therapy-refractory CNS aspergillosis when added to systemic therapy., (Copyright © 2020 The Author(s). Published by Elsevier Ltd.. All rights reserved.)

Published
2020
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50. Correction: Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT).

Author

Cesaro S, Pillon M, Sauer M, Smiers F, Faraci M, de Heredia CD, Wynn R, Greil J, Locatelli F, Veys P, Uyttebroeck A, Ljungman P, Chevalier P, Ansari M, Badell I, Güngör T, Salim R, Tischer J, Tecchio C, Russell N, Chybicka A, Styczynski J, Krivan G, Smith O, Stein J, Afanasyev B, Pochon C, Menconi MC, Bosman P, Mauro M, Tridello G, de Latour RP, and Dufour C

Abstract

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Published
2020
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