Hematopoietic Stem Cell Transplantation for Shwachman-Diamond Syndrome
| Autor: | Mary Eapen, Rammurti T. Kamble, Margaret L. MacMillan, Kyle Hebert, Joseph H. Antin, Lauri Burroughs, Kasiani C. Myers, Farid Boulad, Inga Hofmann |
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| Rok vydání: | 2020 |
| Předmět: |
Oncology
medicine.medical_specialty Transplantation Conditioning medicine.medical_treatment Graft vs Host Disease Hematopoietic stem cell transplantation Article 03 medical and health sciences Myelogenous 0302 clinical medicine hemic and lymphatic diseases Internal medicine medicine Humans Transplantation Homologous Aplastic anemia Cause of death Transplantation Cytopenia business.industry Hematopoietic Stem Cell Transplantation Bone marrow failure Hematology medicine.disease Shwachman-Diamond Syndrome Leukemia surgical procedures operative 030220 oncology & carcinogenesis Unrelated Donors business 030215 immunology |
| Zdroj: | Biol Blood Marrow Transplant |
| ISSN: | 1083-8791 |
| Popis: | We report the outcomes of hematopoietic stem cell transplantation (HSCT) for 52 patients with Shwachman-Diamond syndrome (SDS) who underwent transplantation between 2000 and 2017. The median age at transplantation was 11 years, and the median duration of follow-up was 60 months. The indication for HSCT was bone marrow failure (BMF; cytopenia or aplastic anemia) in 39 patients and myelodysplasia (MDS)/acute myelogenous leukemia (AML) in 13 patients. The donor type was an HLA-matched sibling for 18 patients, an HLA-matched or mismatched relative for 6 patients, and an HLA-matched or mismatched unrelated donor for 28 patients. Preparative regimens for BMF were myeloablative in 13 patients and reduced intensity in 26. At the time of this report, 29 of the 39 patients with BMF were alive, and the 5-year overall survival was 72% (95% confidence interval, 57% to 86%). Graft failure and graft-versus-host disease were the predominant causes of death. Preparative regimens for patients with MDS/AML were myeloablative in 8 and reduced intensity in 5. At the time of this report, only 2 of 13 patients were alive (15%), with relapse the predominant cause of death. Survival after transplantation for SDS-related BMF is better compared with historical reports, but strategies are needed to overcome graft failure and graft-versus-host disease. For SDS- related MDS or AML, transplantation does not extend survival. Rigorous surveillance and novel treatments for leukemia are urgently needed. |
| Databáze: | OpenAIRE |
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