ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2020
Hematopoietic Stem Cell Transplantation for Shwachman-Diamond Syndrome.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed, 35 citations in OpenAlex.
- Outcomes of germ line testing for children with hematologic malignancies undergoing hematopoietic cell transplantation.Blood advances · 2026Article
- Endoscopic findings in patients with Shwachman-Diamond syndrome: A report from the North American Shwachman-Diamond syndrome registry.JPGN reports · 2026Article
- Liver disease in Shwachman-Diamond syndrome: A comprehensive characterization across the age spectrum.Journal of pediatric gastroenterology and nutrition · 2026Article
- Bringing the rare into sight: applications of haploidentical stem cell transplantation in the treatment of rare diseases.Bone marrow transplantation · 2026Review
- Growth Patterns in Shwachman-Diamond Syndrome: Findings from the North American Shwachman-Diamond Syndrome Registry.The Journal of pediatrics · 2025Article
- Genetic and clinical characteristics of patients with Shwachman Diamond syndrome with special consideration of treatment with granulocyte-colony stimulating factor.Haematologica · 2025Article
- Case Report: A case report and literature review of shwachman-diamond syndrome concurrent with klinefelter syndrome.Frontiers in pediatrics · 2025Article
- Clinical characteristics and genetic mutation analysis in 18 pediatric patients with Shwachman-Diamond syndrome.Frontiers in genetics · 2025Article
- [Allogeneic hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: a report of three cases and literature review].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2024Review
- Article
- Pediatric Bone Marrow Failure: A Broad Landscape in Need of Personalized Management.Journal of clinical medicine · 2023Review
- Spectrum of diabetes mellitus in patients with Shwachman-Diamond syndrome: case report and review of the literature.Italian journal of pediatrics · 2023Review
- Review
- Genetic testing in severe aplastic anemia is required for optimal hematopoietic cell transplant outcomes.Blood · 2022Article
- Recent advances in hematopoietic cell transplantation for inherited bone marrow failure syndromes.International journal of hematology · 2022Review
- Identification of an asymptomatic Shwachman-Bodian-Diamond syndrome mutation in a patient with acute myeloid leukemia.International journal of hematology · 2022Article
- Outcomes in Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia.Transplantation and cellular therapy · 2022Article
- Inducible Sbds deletion impairs bone marrow niche capacity to engraft donor bone marrow after transplantation.Blood advances · 2022Article
- Hematologic complications with age in Shwachman-Diamond syndrome.Blood advances · 2022Article
- Case report: Venetoclax therapy in a boy with acute myeloid leukemia in Shwachman Diamond syndrome.Frontiers in pediatrics · 2022Article
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Authors and funding
9 authors at 8 institutions in 1 country.
Funding
Abstract
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.
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