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7 results on '"Kalwak, Krzysztof"'

1. Advanced Phases at Diagnosis of Childhood Chronic Myeloid Leukemia : The Experience of the International Registry for Chronic Myeloid Leukemia (CML) in Children and Adolescents (I-CML-Ped Study)

Author

Millot, Frederic, Guilhot, Joelle, Suttorp, Meinolf, Maledon, Natacha, Adalet, Meral Gunes, Sedlacek, Petr, Bont, Eveline S., Li, Chi Kong, Kalwak, Krzysztof, Lausen, Birgitte, Jakovljevic, Gordana, Dworzak, Michael, Hraskova, Andrea, Moerloose, Barbara, roula farah, Biondi, Andrea, and Baruchel, Andre

2. ACUTE AND CHRONIC GVHD ARE DIFFERENTLY INFLUENCED BY THE LEVEL OF HLA ALLELIC AND HAPLOTYPIC DISPARITY: A POLISH IMMUNOGENETICS STUDY GROUP

Author

Mika-Witkowska, Renata, Rogatko-Koros, Marta, Graczyk-Pol, Elzbieta, Marosz-Rudnicka, Anna, Dlugokecka, Agnieszka, Lange, Andrzej, Mizia, Sylwia, Bogunia-Kubik, Katarzyna, Wysoczanska, Barbara, Markiewicz, Miroslaw, Koclega, Anna, Kyrcz-Krzemien, Slawomira, Baranska, Malgorzata, Wachowiak, Jacek, Gil, Lidia, Czyz, Anna, Lojko, Anna, Komarnicki, Mieczyslaw, Mielcarek, Monika, Kalwak, Krzysztof, Tomaszewska, Agnieszka, Nasilowska-Adamska, Barbara, Szczepinski, Andrzej, Marianska, Bozena, Halaburda, Kazimierz, Gronkowska, Anna, Jedrzejczak, Wieslaw W., Styczynski, Jan, Bieniaszewska, Maria, Kucharska, Anna, Hellmann, Andrzej, Dybko, Jaroslaw, Kuliczkowski, Kazimierz, Drabko, Katarzyna, Kowalczyk, Jerzy, Czerw, Tomasz, Giebel, Sebastian, Holowiecki, Jerzy, Gozdzik, Jolanta, Malinowska, Agnieszka, Dziopa, Joanna, Szlendak, Urszula, Gawron, Agnieszka, Czyz, Jaroslaw, Warzocha, Krzysztof, and Jacek Nowak

4. Results of a clinical phase II trial of treosulfan/fludarabine-based conditioning therapy prior to allogeneic haematopoietic stem cell transplantation in paediatric patients with haematological malignancies

Author

Kalwak, Krzysztof, Monika Mielcarek-Siedziuk, Patrick, Katharine, Styczynski, Jan, Bader, Peter, Corbacioglu, Selim, Burkhardt, Birgit, Sykora, Karl-Walter, Drabko, Katarzyna, Gozdzik, Jolanta, Fagioli, Franca, Greil, Johann, Gruhn, Bernd, Kremens, Bernhard, Locatelli, Franco, Mueller, Ingo, Schlegel, Paul-Gerhardt, Sedlacek, Petr, Stachel, Klaus Daniel, and Vora, Ajay

Subjects
Medizin

5. HLA INFERRED MHC HAPLOTYPE DISPARITY BETTER PREDICTED HEMATOPOIETIC STEM CELL TRANSPLANT OUTCOME THAN HLA ALLELE/ANTIGEN DISPARITY IN STANDARD RISK RECIPIENTS: A POLISH IMMUNOGENETICS STUDY GROUP

Author

Graczyk-Pol, Elzbieta, Marosz-Rudnicka, Anna, Mika-Witkowska, Renata, Dlugokecka, Agnieszka, Rogatko-Koros, Marta, Lange, Andrzej, Mizia, Sylwia, Bogunia-Kubik, Katarzyna, Wysoczanska, Barbara, Markiewicz, Miroslaw, Koclega, Anna, Kyrcz-Krzemien, Slawomira, Baranska, Malgorzata, Wachowiak, Jacek, Gil, Lidia, Czyz, Anna, Lojko, Anna, Komarnicki, Mieczyslaw, Monika Mielcarek-Siedziuk, Kalwak, Krzysztof, Tomaszewska, Agnieszka, Nasilowska-Adamska, Barbara, Szczepinski, Andrzej, Marianska, Bozena, Halaburda, Kazimierz, Gronkowska, Anna, Jedrzejczak, Wieslaw W., Styczynski, Jan, Bieniaszewska, Maria, Kucharska, Anna, Hellmann, Andrzej, Dybko, Jaroslaw, Kuliczkowski, Kazimierz, Drabko, Katarzyna, Kowalczyk, Jerzy, Czerw, Tomasz, Giebel, Sebastian, Holowiecki, Jerzy, Gozdzik, Jolanta, Malinowska, Agnieszka, Dziopa, Joanna, Szlendak, Urszula, Gawron, Agnieszka, Czyz, Jaroslaw, Warzocha, Krzysztof, and Nowak, Jacek

7. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study

Author

Barzaghi, F., Hernandez, L.C.A., Neven, B., Ricci, S., Kucuk, Z.Y., Bleesing, J.J., Nademi, Z., Slatter, M.A., Ulloa, E.R., Shcherbina, A., Roppelt, A., Worth, A., Silva, J., Aiuti, A., Murguia-Favela, L., Speckmann, C., Carneiro-Sampaio, M., Fernandes, J.F., Baris, S., Ozen, A., Karakoc-Aydiner, E., Kiykim, A., Schulz, A., Steinmann, S., Notarangelo, L.D., Gambineri, E., Lionetti, P., Shearer, W.T., Forbes, L.R., Martinez, C., Moshous, D., Blanche, S., Fisher, A., Ruemmele, F.M., Tissandier, C., Ouachee-Chardin, M., Rieux-Laucat, F., Cavazzana, M., Qasim, W., Lucarelli, B., Albert, M.H., Kobayashi, I., Alonso, L., Heredia, C.D. de, Kanegane, H., Lawitschka, A., Seo, J.J., Gonzalez-Vicent, M., Diaz, M.A., Goyal, R.K., Sauer, M.G., Yesilipek, A., Kim, M., Yilmaz-Demirdag, Y., Bhatia, M., Khlevner, J., Padilla, E.J.R., Martino, S., Montin, D., Neth, O., Molinos-Quintana, A., Valverde-Fernandez, J., Broides, A., Pinsk, V., Ballauf, A., Haerynck, F., Bordon, V., Dhooge, C., Garcia-Lloret, M.L., Bredius, R.G., Kawak, K., Haddad, E., Seidel, M.G., Duckers, G., Pai, S.Y., Dvorak, C.C., Ehl, S., Locatelli, F., Goldman, F., Gennery, A.R., Cowan, M.J., Roncarolo, M.G., Bacchetta, R., PIDTC, IEWP, European Soc Blood Marrow, Barzaghi, Federica, Hernandez, Laura Cristina Amaya, Neven, Benedicte, Ricci, Silvia, Kucuk, Zeynep Yesim, Bleesing, Jack J., Nademi, Zohreh, Slatter, Mary Anne, Ulloa, Erlinda Rose, Shcherbina, Anna, Roppelt, Anna, Worth, Austen, Silva, Juliana, Aiuti, Alessandro, Murguia-Favela, Luis, Speckmann, Carsten, Carneiro-Sampaio, Magda, Fernandes, Juliana Folloni, Baris, Safa, Ozen, Ahmet, Karakoc-Aydiner, Elif, Kiykim, Ayca, Schulz, Ansgar, Steinmann, Sandra, Notarangelo, Lucia Dora, Gambineri, Eleonora, Lionetti, Paolo, Shearer, William Thomas, Forbes, Lisa R., Martinez, Caridad, Moshous, Despina, Blanche, Stephane, Fisher, Alain, Ruemmele, Frank M., Tissandier, Come, Ouachee-Chardin, Marie, Rieux-Laucat, Frederic, Cavazzana, Marina, Qasim, Waseem, Lucarelli, Barbarella, Albert, Michael H., Kobayashi, Ichiro, Alonso, Laura, De Heredia, Cristina Diaz, Kanegane, Hirokazu, Lawitschka, Anita, Seo, Jong Jin, Gonzalez-Vicent, Marta, Diaz, Miguel Angel, Goyal, Rakesh Kumar, Sauer, Martin G., Yesilipek, Akif, Kim, Minsoo, Yilmaz-Demirdag, Yesim, Bhatia, Monica, Khlevner, Julie, Padilla, Erick J. Richmond, Martino, Silvana, Montin, Davide, Neth, Olaf, Molinos-Quintana, Agueda, Valverde-Fernandez, Justo, Broides, Arnon, Pinsk, Vered, Ballauf, Antje, Haerynck, Filomeen, Bordon, Victoria, Dhooge, Catharina, Garcia-Lloret, Maria Laura, Bredius, Robbert G., Kalwak, Krzysztof, Haddad, Elie, Seidel, Markus Gerhard, Duckers, Gregor, Pai, Sung-Yun, Dvorak, Christopher C., Ehl, Stephan, Locatelli, Franco, Goldman, Frederick, Gennery, Andrew Richard, Cowan, Mort J., Roncarolo, Maria-Grazia, Bacchetta, Rosa, Amaya Hernandez, Laura Cristina, Murguia-Favela, Lui, Shearer, William Thoma, Rieux-Laucat, Frédéric, Diaz De Heredia, Cristina, Richmond Padilla, Erick J., and Kałwak, Krzysztof

Subjects
0301 basic medicine, Male, Allergy, medicine.medical_treatment, Medizin, Disease, Hematopoietic stem cell transplantation, SIROLIMUS, Regenerative Medicine, primary immune deficiency, Medicine and Health Sciences, IPEX, Immunology and Allergy, 2.1 Biological and endogenous factors, Enteropathy, Aetiology, POLYENDOCRINOPATHY, Child, Pediatric, CÉLULAS-TRONCO, immunosuppression, Hematopoietic Stem Cell Transplantation, Genetic Diseases, X-Linked, Immunosuppression, Forkhead Transcription Factors, X-LINKED SYNDROME, Allografts, Survival Rate, surgical procedures, operative, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, Immune System Diseases, Genetic Diseases, Child, Preschool, hematopoietic stem cell transplantation, Female, hematopoietic stem, neonatal diabetes, FOXP3, Primary Immune Deficiency, Treg cells, enteropathy, genetic autoimmunity, rapamycin, Type 1, Diarrhea, Adult, medicine.medical_specialty, Adolescent, Immunology, Neonatal onset, Article, Disease-Free Survival, 03 medical and health sciences, Neonatal diabete, Clinical Research, Internal medicine, IMMUNODYSREGULATION, medicine, Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation, Diabetes Mellitus, Genetics, Humans, REGULATORY T-CELLS, cell transplantation, Preschool, Retrospective Studies, Immunosuppression Therapy, Transplantation, IMMUNE DYSREGULATION, business.industry, MUTATIONS, Infant, Retrospective cohort study, STEM-CELL TRANSPLANTATION, IPEX syndrome, X-Linked, medicine.disease, Stem Cell Research, BONE-MARROW-TRANSPLANTATION, Treg cell, FOXP3 MUTATIONS, Diabetes Mellitus, Type 1, 030104 developmental biology, ENGRAFTMENT, Mutation, business, Follow-Up Studies
Abstract

Background Immunodysregulation polyendocrinopathy enteropathy x-linked(IPEX) syndromeis a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined. Objective This analysis sought to evaluate disease onset, progression, and long-term outcome of the 2 main treatments in long-term IPEX survivors. Methods Clinical histories of 96 patients with a genetically proven IPEX syndrome were collected from 38 institutions worldwide and retrospectively analyzed. To investigate possible factors suitable to predict the outcome, an organ involvement (OI) scoring system was developed. Results We confirm neonatal onset with enteropathy, type 1 diabetes, and eczema. In addition, we found less common manifestations in delayed onset patients or during disease evolution. There is no correlation between the site of mutation and the disease course or outcome, and the same genotype can present with variable phenotypes. HSCT patients (n = 58) had a median follow-up of 2.7 years (range, 1 week-15 years). Patients receiving chronic IS (n = 34) had a median follow-up of 4 years (range, 2 months-25 years). The overall survival after HSCT was 73.2% (95% CI, 59.4-83.0) and after IS was 65.1% (95% CI, 62.8-95.8). The pretreatment OI score was the only significant predictor of overall survival after transplant (P = .035) but not under IS. Conclusions Patients receiving chronic IS were hampered by disease recurrence or complications, impacting long-term disease-free survival. When performed in patients with a low OI score, HSCT resulted in disease resolution with better quality of life, independent of age, donor source, or conditioning regimen., GRAPHICAL ABSTRACT

Published
2018

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