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1. Knochenmark

Author

Sykora, K.-W., Welte, K., Niemeyer, C., Handgretinger, R., Matthes-Martin, S., Lang, P., Strahm, B., Kratz, C., Niemeyer, Charlotte, editor, and Eggert, Angelika, editor

Published
2018
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12. Second allogeneic hematopoietic stem cell transplantation (HSCT) results in outcome similar to that of first HSCT for patients with juvenile myelomonocytic leukemia

Author

Yoshimi, A, Mohamed, M, Bierings, M, Urban, C, Korthof, E, Zecca, M, Sykora, K-W, Duffner, U, Trebo, M, Matthes-Martin, S, Sedlacek, P, Klingebiel, T, Lang, P, Führer, M, Claviez, A, Wössmann, W, Pession, A, Arvidson, J, O'Marcaigh, A S, van den Heuvel-Eibrink, M M, Starý, J, Hasle, H, Nöllke, P, Locatelli, F, and Niemeyer, C M

Published
2007
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40. Sickle cell disease: An international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

Author

Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Simoes, B. P., Ferster, A., Dupont, S., De La Fuente, J., Dalle, J. -H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., Kuentz, M., Michel, G., Apperley, J., Lutz, P., Neven, B., Bertrand, Y., Vannier, J. P., Ayas, M., Cavazzana, M., Matthes-Martin, S., Rocha, V., Elayoubi, H., Kenzey, C., Bader, P., Locatelli, Franco, Ruggeri, A., Eapen, M., Bordon, V., Labarque, V., Pereira, M., Bittencourt, H., Petersen, H., Deconninck, E., Jubert, C., Perrin, J., Cahn, J. Y., Bruno, B., Bordigoni, P., Mechinaud, F., Vernant, J. P., Stephan, J. L., Suttorp, M., Strahm, B., Da Cunha, C. B., Garwer, B., Rothmayer, M., Wendelin, K., Graphakos, S., Tbakhi, A., Naeimi, N., Zuckerman, T., Sharon, P. B., Yaniv, I., Amos, T., Prete, A., Lo Nigro, L., Lanino, E., Faraci, M., Ciceri, F., Marktel, S., De Simone, G., Messina, C., Bartolomeo, P. D. I., Santarone, S., Vallisa, D., Bertaina, A., Arcese, W., Foa, R., Berger, M., Maximova, N., Wallet, S., Bazuaye, G. N., Maschan, A., De Heredia, C. D., Bieler, C. B., Pato, J. R., Heras, I., Trevor, R., Abayomi, K., Thomson, J., Fasth, A., Frodin, U., Ljugman, P., Ansari, M., Gungor, T., Unal, E., Pehlivan, M., Anak, S., Ozturk, G., Unal, A., Lawson, S., Keshani, J., Drake, A., Wynn, R., Williams, J., Jagsia, M., Leung, W., Abraham, A., Sahdey, I., Margolis, D., Eames, G., Horwitz, E., Cowan, M., Kapoor, N., Rowley, S., Megason, G., Rogers, Z., Bolanos-Meade, J., Hudspeth, M., Rosenthal, J., Olson, T., Kassow, K., Selby, G., Haines, H., Chaudhury, S., Gluckman, E., Cappelli, B., Bernaudin, F., Labopin, M., Volt, F., Carreras, J., Simoes, B. P., Ferster, A., Dupont, S., De La Fuente, J., Dalle, J. -H., Zecca, M., Walters, M. C., Krishnamurti, L., Bhatia, M., Leung, K., Yanik, G., Kurtzberg, J., Dhedin, N., Kuentz, M., Michel, G., Apperley, J., Lutz, P., Neven, B., Bertrand, Y., Vannier, J. P., Ayas, M., Cavazzana, M., Matthes-Martin, S., Rocha, V., Elayoubi, H., Kenzey, C., Bader, P., Locatelli, F., Ruggeri, A., Eapen, M., Bordon, V., Labarque, V., Pereira, M., Bittencourt, H., Petersen, H., Deconninck, E., Jubert, C., Perrin, J., Cahn, J. Y., Bruno, B., Bordigoni, P., Mechinaud, F., Vernant, J. P., Stephan, J. L., Suttorp, M., Strahm, B., Da Cunha, C. B., Garwer, B., Rothmayer, M., Wendelin, K., Graphakos, S., Tbakhi, A., Naeimi, N., Zuckerman, T., Sharon, P. B., Yaniv, I., Amos, T., Prete, A., Lo Nigro, L., Lanino, E., Faraci, M., Ciceri, F., Marktel, S., De Simone, G., Messina, C., Bartolomeo, P. D. I., Santarone, S., Vallisa, D., Bertaina, A., Arcese, W., Foa, R., Berger, M., Maximova, N., Wallet, S., Bazuaye, G. N., Maschan, A., De Heredia, C. D., Bieler, C. B., Pato, J. R., Heras, I., Trevor, R., Abayomi, K., Thomson, J., Fasth, A., Frodin, U., Ljugman, P., Ansari, M., Gungor, T., Unal, E., Pehlivan, M., Anak, S., Ozturk, G., Unal, A., Lawson, S., Keshani, J., Drake, A., Wynn, R., Williams, J., Jagsia, M., Leung, W., Abraham, A., Sahdey, I., Margolis, D., Eames, G., Horwitz, E., Cowan, M., Kapoor, N., Rowley, S., Megason, G., Rogers, Z., Bolanos-Meade, J., Hudspeth, M., Rosenthal, J., Olson, T., Kassow, K., Selby, G., Haines, H., Chaudhury, S., France Monacord, Centre Scientifique de Monaco (CSM), CHI Créteil, Centre de Recherche Saint-Antoine (CR Saint-Antoine), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC), Eurocord, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Institut Universitaire d'Hématologie (IUH), Université Paris Diderot - Paris 7 (UPD7)-Université Paris Diderot - Paris 7 (UPD7), Hôpital Universitaire des Enfants - Reine Fabiola, Université Libre de Bruxelles, Equipe NEMESIS - Centre de Recherches de l'Institut du Cerveau et de la Moelle épinière (NEMESIS-CRICM), Centre de Recherche de l'Institut du Cerveau et de la Moelle épinière (CRICM), Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Centre National de la Recherche Scientifique (CNRS)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Pierre et Marie Curie - Paris 6 (UPMC), Unité d'Hémato-Immunologie pédiatrique [Hôpital Robert Debré, Paris], Service d'Immuno-hématologie pédiatrique [Hôpital Robert Debré, Paris], Hôpital Robert Debré-Hôpital Robert Debré, Duke University Medical Center, Service de Greffe de Moelle - Unité AJA, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (APHP), Service d'hématologie clinique, Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Hôpital de la Timone [CHU - APHM] (TIMONE), Department of Haematology, Hôpital Civil, Hopital Civil, Département d'Immunologie, hématologie et rhumatologie pédiatriques [Hôpital Necker-Enfants malades - APHP], CHU Necker - Enfants Malades [AP-HP], Centre Hospitalier Lyon Sud [CHU - HCL] (CHLS), Hospices Civils de Lyon (HCL), Hemostase, Endothelium, Angiogenese (UMR_S_553), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Diderot - Paris 7 (UPD7), Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER), Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Imagine - Institut des maladies génétiques (IMAGINE - U1163), Centre National de la Recherche Scientifique (CNRS)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Churchill Hospital [Breast Care Unit], Churchill Hospital Oxford Centre for Haematology, Santa Lucia Foundation, IRCSS, Rome, Medical College of Wisconsin, National Institute for Health Research, Centre de Recherche Saint-Antoine (UMRS893), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut Universitaire d'Hématologie (IUH), Hôpital Universitaire des Enfants Reine Fabiola [Bruxelles, Belgique] (HUDERF), Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Groupe Hospitalier Saint Louis - Lariboisière - Fernand Widal [Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Université Paris Diderot - Paris 7 (UPD7)-Institut National de la Santé et de la Recherche Médicale (INSERM), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, and UCL - (SLuc) Service d'hématologie et d'oncologie pédiatrique

Subjects
Male, HYDROXYUREA, Transplantation Conditioning, [SDV]Life Sciences [q-bio], medicine.medical_treatment, CHILDREN, Hematopoietic stem cell transplantation, Biochemistry, THALASSEMIA, 0302 clinical medicine, HLA Antigens, Surveys and Questionnaires, 1114 Paediatrics And Reproductive Medicine, Child, ComputingMilieux_MISCELLANEOUS, CÉLULAS-TRONCO, Hazard ratio, Graft Survival, BONE-MARROW TRANSPLANT, Hematopoietic Stem Cell Transplantation, Hematology, Sickle cell anemia, 3. Good health, Survival Rate, medicine.anatomical_structure, Treatment Outcome, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, 030220 oncology & carcinogenesis, Child, Preschool, Histocompatibility, [SDV.IMM]Life Sciences [q-bio]/Immunology, Female, Life Sciences & Biomedicine, medicine.medical_specialty, Adolescent, Anemia, Immunology, Anemia, Sickle Cell, 1102 Cardiovascular Medicine And Haematology, Disease-Free Survival, 03 medical and health sciences, Internal medicine, MANAGEMENT, medicine, Humans, ANEMIA, Survival rate, Transplantation, Science & Technology, business.industry, Siblings, Sickle cell disease, Infant, 1103 Clinical Sciences, ADULTS, Cell Biology, medicine.disease, LIFE, EXPERT PANEL, Bone marrow, FOLLOW-UP, business, 030215 immunology
Abstract

Despite advances in supportive therapy to prevent complications of sickle cell disease (SCD), access to care is not universal. Hematopoietic cell transplantation is, to date, the only curative therapy for SCD, but its application is limited by availability of a suitable HLA-matched donor and lack of awareness of the benefits of transplant. Included in this study are 1000 recipients of HLA-identical sibling transplants performed between 1986 and 2013 and reported to the European Society for Blood and Marrow Transplantation, Eurocord, and the Center for International Blood and Marrow Transplant Research. The primary endpoint was event-free survival, defined as being alive without graft failure; risk factors were studied using a Cox regression models. The median age at transplantation was 9 years, and the median follow-up was longer than 5 years. Most patients received a myeloablative conditioning regimen (n = 873; 87%); the remainder received reduced-intensity conditioning regimens (n = 125; 13%). Bone marrow was the predominant stem cell source (n = 839; 84%); peripheral blood and cord blood progenitors were used in 73 (7%) and 88 (9%) patients, respectively. The 5-year event-free survival and overall survival were 91.4% (95% confidence interval, 89.6%-93.3%) and 92.9% (95% confidence interval, 91.1%-94.6%), respectively. Event-free survival was lower with increasing age at transplantation (hazard ratio [HR], 1.09; P < .001) and higher for transplantations performed after 2006 (HR, 0.95; P = .013). Twenty-three patients experienced graft failure, and 70 patients (7%) died, with the most common cause of death being infection. The excellent outcome of a cohort transplanted over the course of 3 decades confirms the role of HLA-identical sibling transplantation for children and adults with SCD.

Published
2017

41. Two pregnancies shortly after transplantation with reduced intensity conditioning in chronic myeloid leukemia

Author

Faraci, M, Matthes-Martin, S, Lanino, E, Morreale, G, Ferretti, M, Giardino, S, Micalizzi, C, Balduzzi, A, Faraci M, Matthes-Martin S, Lanino E, Morreale G, Ferretti M, Giardino S, Micalizzi C, Balduzzi A, Faraci, M, Matthes-Martin, S, Lanino, E, Morreale, G, Ferretti, M, Giardino, S, Micalizzi, C, Balduzzi, A, Faraci M, Matthes-Martin S, Lanino E, Morreale G, Ferretti M, Giardino S, Micalizzi C, and Balduzzi A

Abstract

POI is a relevant late complication after HSCT and occurring more frequently after MAC than after RIC regimens. Reports on the frequency of POI after RIC in a large pediatric and adolescent population are lacking. In this study, we describe a girl affected by CML diagnosed at the age of 15 yr and treated with oncarbide and interferon followed by imatinib and dasatinib. She had two pregnancies shortly after RIC performed according to the CML-SCT I-BFM protocol including TT, FLU, and MEL. Hypergonadotropic hypogonadism occurred four months after HSCT; menstruations resumed regularly six months after HSCT. Eight and 20 months after HSCT, the patient became pregnant and then delivered, respectively, two babies at term by cesarean section. Both newborns had no neonatal complications. Donor chimerism at time of two pregnancies and five yr after transplantation demonstrated complete donor engraftment. These findings suggest that I-BFM CML-SCT protocol could be a promising treatment option for adolescents or young adults with CML eligible for HSCT.

Published
2016

44. Stem cell transplantation in severe congenital neutropenia: An analysis from the European Society for Blood and Marrow Transplantation

Author

Fioredda, F., Iacobelli, S., Biezen, A. van, Gaspar, B., Ancliff, P., Donadieu, J., Aljurf, M., Peters, C., Calvillo, M., Matthes-Martin, S., Morreale, G., van't Veer-Tazelaar, N., Wreede, L. de, Seraihy, A. al, Yesilipek, A., Fischer, A., Bierings, M., Ozturk, G., Smith, O., Veys, P., Ljungman, P., Latour, R.P. de, Codina, J.S.D., Or, R., Ganser, A., Afanasyev, B., Wynn, R., Kalwak, K., Marsh, J., Dufour, C., Pediat Dis Working Parties Europea, Fioredda, Francesca, Iacobelli, Simona, Van Biezen, Anja, Gaspar, Bobby, and Risitano, ANTONIO MARIA

Subjects
Male, Graft Rejection, medicine.medical_treatment, Graft vs Host Disease, Hematopoietic stem cell transplantation, Biochemistry, Allograft, Retrospective Studie, Cumulative incidence, Non-U.S. Gov't, Child, Societies, Medical, Incidence (epidemiology), Research Support, Non-U.S. Gov't, Histocompatibility Testing, Incidence, Medicine (all), Hematopoietic Stem Cell Transplantation, Hematology, Allografts, Clinical Trial, Multicenter Study, Europe, surgical procedures, operative, Child, Preschool, Acute Disease, Cyclosporine, Female, Unrelated Donors, Kostmann syndrome, Human, Adult, medicine.medical_specialty, Neutropenia, Adolescent, Immunology, Research Support, Settore MED/01 - Statistica Medica, Middle East, Internal medicine, Medical, medicine, Journal Article, Humans, Aplastic anemia, Congenital Neutropenia, Preschool, Retrospective Studies, business.industry, Unrelated Donor, Cell Biology, medicine.disease, Surgery, Transplantation, Methotrexate, business, Societies
Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment of severe congenital neutropenia (SCN), but data on outcome are scarce. We report on the outcome of 136 SCN patients who underwent HSCT between 1990 and 2012 in European and Middle East centers. The 3-year overall survival (OS) was 82%, and transplant-related mortality (TRM) was 17%. In multivariate analysis, transplants performed under the age of 10 years, in recent years, and from HLA-matched related or unrelated donors were associated with a significantly better OS. Frequency of graft failure was 10%. Cumulative incidence (day +90) of acute graft-versus-host disease (GVHD) grade 2-4 was 21%. In multivariate analysis, HLA-matched related donor and prophylaxis with cyclosporine A and methotrexate were associated with lower occurrence of acute GVHD. Cumulative incidence (1 year) of chronic GVHD was 20%. No secondary malignancies occurred after a median follow-up of 4.6 years. These data show that the outcome of HSCT for SCN from HLA-matched donors, performed in recent years, in patients younger than 10 years is acceptable. Nevertheless, given the TRM, a careful selection of HSCT candidates should be undertaken.

Published
2015

45. Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents

Author

Wlodarski, M. W., Hirabayashi, S., Pastor, V., Stary, J., Hasle, H., Masetti, R., Dworzak, M., Schmugge, M., Van Den Heuvel-Eibrink, M., Ussowicz, M., De Moerloose, B., Catala, A., Smith, O. P., Sedlacek, P., Lankester, A. C., Zecca, M., Bordon, V., Matthes-Martin, S., Abrahamsson, J., Kuhl, J. S., Sykora, K. -W., Albert, M. H., Przychodzien, B., Maciejewski, J. P., Schwarz, S., Gohring, G., Schlegelberger, B., Cseh, A., Noellke, P., Yoshimi, A., Locatelli, Franco, Baumann, I., Strahm, B., Niemeyer, C. M., Locatelli F. (ORCID:0000-0002-7976-3654), Wlodarski, M. W., Hirabayashi, S., Pastor, V., Stary, J., Hasle, H., Masetti, R., Dworzak, M., Schmugge, M., Van Den Heuvel-Eibrink, M., Ussowicz, M., De Moerloose, B., Catala, A., Smith, O. P., Sedlacek, P., Lankester, A. C., Zecca, M., Bordon, V., Matthes-Martin, S., Abrahamsson, J., Kuhl, J. S., Sykora, K. -W., Albert, M. H., Przychodzien, B., Maciejewski, J. P., Schwarz, S., Gohring, G., Schlegelberger, B., Cseh, A., Noellke, P., Yoshimi, A., Locatelli, Franco, Baumann, I., Strahm, B., Niemeyer, C. M., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

GermlineGATA2 mutations cause cellular deficiencieswith high propensity for myeloid disease. We investigated 426 children and adolescents with primary myelodysplastic syndrome (MDS) and 82 cases with secondary MDS enrolled in 2 consecutive prospective studies of the European Working Group of MDS in Childhood (EWOGMDS) conducted in Germany over a period of 15 years. Germline GATA2 mutations accounted for 15% of advanced and 7% of all primary MDS cases, but were absent in children with MDS secondary to therapy or acquired aplastic anemia. Mutation carriers were older at diagnosis and more likely to present with monosomy 7 and advanced disease compared with wild-type cases. For stratified analysis according to karyotype, 108 additional primary MDS patients registered with EWOG-MDS were studied. Overall, we identified 57 MDS patients with germline GATA2mutations. GATA2 mutations were highly prevalent among patients with monosomy 7 (37%, all ages) reaching its peak in adolescence (72%of adolescents withmonosomy 7).Unexpectedly, monocytosis was more frequent in GATA2-mutated patients. However, when adjusted for the selection bias from monosomy 7, mutational status had no effect on the hematologic phenotype. Finally, overall survival and outcome of hematopoietic stem cell transplantation (HSCT) were not influenced by mutational status. This study identifies GATA2 mutations as the most common germline defect predisposing to pediatric MDS with a very high prevalence in adolescents with monosomy 7. GATA2 mutations do not confer poor prognosis in childhood MDS. However, the high risk for progression to advanced diseasemust guide decision-making toward timely HSCT.

Published
2016

46. HAEMATOPOIETIC STEM CELL TRANSPLANTATION IN SEVERE CONGENITAL NEUTROPENIA: DATA FROM THE EUROPEAN GROUP FOR BLOOD AND MARROW TRANSPLANTATION

Author

Fioredda, F., Wreede, L. C., Biezen, A., T Veer-Tazelaar, N., Michaela Calvillo, Gaspar, B., Aljurf, M., Peters, C., Yesilipek, A., Fisher, A., Bierings, M., Donadieu, J., Ozturk, G., Ljungman, P., Or, R., Ganser, A., Veys, P., Matthes-Martin, S., Kalwak, K., Wynn, R., Afanasyev, B., Toledo Codina, J. S., Marsh, J., and Dufour, C.

Published
2014

47. 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

48. MANAGEMENT OF DOCK8 DEFICIENCY BY HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT)

Author

Aydin, S., Freeman, A. F., Sanal, O., Al-Mousa, H., Matthes-Martin, S., Cuellar-Rodriguez, J., Hickstein, D. D., Tavil, B., Azik, F. M., Bittner, T. C., Bredius, R. G., Ayvaz, D., Kuskonmaz, B., Hoenig, M., Schulz, A., Picard, C., Barlogis, V., Gennery, A., Ifversen, M., Montfrans, J. M., Kuijpers, T. W., Dueckers, G., Al-Herz, W., Pai, S. Y., Geha, R. S., Notheis, G., Schwarze, C. P., Schuster, F., Gathmann, B., Grimbacher, B., Gaspar, B., Belohradsky, B. H., Ochs, H., Ellen Renner, Chatila, T., Engelhardt, K. R., and Albert, M. H.

Published
2012

49. Long-term outcome following hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and the European Group for Blood and Marrow Transplantation

Author

Ozsahin, H, CAVAZZANA CALVO, M, Notarangelo, Luigi Daniele, Schulz, A, Thrasher, Aj, Mazzolari, Evelina, Slatter, Ma, LE DEIST, F, Blanche, S, Veys, P, Fasth, A, Bredius, R, Sedlacek, P, Wulfraat, N, Ortega, J, Heilmann, C, O'Meara, A, Wachowiak, J, Kalwak, K, MATTHES MARTIN, S, Gungor, T, Ikinciogullari, A, Landais, P, Cant, Aj, Friederich, W, and Fisher, A.

Published
2007

50. Second allogeneic hematopoietic stem cell transplantation (HSCT) results in outcome similar to that of first HSCT for patients with juvenile myelomonocytic leukemia [4]

Author

Yoshimi, A., Mohamed, M., Bierings, M., Urban, C., Korthof, E., Zecca, M., Sykora, K. -W., Duffner, U., Trebo, M., Matthes-Martin, S., Sedlacek, P., Klingebiel, T., Lang, P., Fuhrer, M., Claviez, A., Wossmann, W., Pession, A., Arvidson, J., O'Marcaigh, A. S., van den Heuvel-Eibrink, M. M., Stary, J., Hasle, H., Nollke, P., Locatelli, F., and Niemeyer, C. M.

Published
2007

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