Diaz-de-Heredia, Cristina and Bresters, Dorine and Faulkner, Lawrence and Yesilipek, Akif and Strahm, Brigitte and Miano, Maurizio and Dalle, Jean-Hugues and Peffault de Latour, Regis and Corbacioglu, Selim (2021) Recommendations on hematopoietic stem cell transplantation for patients with Diamond-Blackfan anemia. On behalf of the Pediatric Diseases and Severe Aplastic Anemia Working Parties of the EBMT. BONE MARROW TRANSPLANTATION, 56 (12). pp. 2956-2963. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
Diamond Blackfan anemia (DBA) is a rare congenital syndrome presenting primarily as pure red cell aplasia with constitutional abnormalities and cancer predisposition. Established treatment options are corticosteroids, regular erythrocyte transfusions with iron chelation therapy, and hematopoietic stem cell transplantation (HSCT). To date, HSCT is the only definitive curative treatment for the hematological phenotype of DBA, but there is little experience with its use. Given the rarity of the disease and its unique features, an expert panel agreed to draw up a set of recommendations on the use of HSCT in DBA to guide clinical decision-making and practice. The recommendations address indications, pretransplant patient evaluation, donor selection, stem cell sources, conditioning regimens, prophylaxis of rejection and graft versus host disease, and post-transplant follow-up.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; CONSORTIUM INTERNATIONAL-CONFERENCE; BLOOD INSTITUTE/PEDIATRIC BLOOD; SYNDROMES CONSENSUS STATEMENT; NATIONAL-CANCER-INSTITUTE; FAILURE SYNDROMES; GROWTH-HORMONE; FERTILITY PRESERVATION; SHORT STATURE; CHILDREN; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 21 Sep 2022 08:31 |
| Last Modified: | 21 Sep 2022 08:31 |
| URI: | https://pred.uni-regensburg.de/id/eprint/47807 |
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