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Outcomes of mismatched and unrelated donor hematopoietic stem cell transplantation in Fanconi anemia conditioned with chemotherapy only
- Source :
- Annals of Hematology. 94:1311-1318
- Publication Year :
- 2015
- Publisher :
- Springer Science and Business Media LLC, 2015.
-
Abstract
- Fanconi anemia (FA) is a genomic instability syndrome associated with bone marrow failure, myelodysplastic syndrome (MDS), and/or acute myeloid leukemia (AML) requiring hematopoietic stem cell transplantation (HSCT) to restore normal hematopoiesis. Although low-intensity fludarabine-based preparative regimens without radiation confer excellent outcomes in FA HSCTs with HLA-matched sibling donors, outcomes for FA patients with alternative donors are less encouraging, albeit improving. We present our experience with 17 FA patients who completed mismatched related or unrelated donor HSCT using a non-radiation fludarabine-based preparative regimen at Charité University Medicine Berlin. All patients engrafted; however, one patient had unstable chimerism in the setting of multi-viral infections that necessitated a stem cell boost to revert to full donor chimerism. Forty-seven percent of patients developed grade I acute graft-verus-host disease (aGVHD). No grade II-IV aGVHD or chronic graft-versus-host disease of any severity occurred. At a median follow-up of 30 months, 88 % of patients are alive with normal hematopoiesis. Two patients died of infections 4 months post-transplantation. These results demonstrate that short-term outcomes for FA patients with mismatched and unrelated donor HSCTs can be excellent using chemotherapy only conditioning. Viral reactivation, however, was a major treatment-related complication.
- Subjects :
- Male
Oncology
medicine.medical_specialty
Transplantation Conditioning
Adolescent
medicine.medical_treatment
Graft vs Host Disease
Antineoplastic Agents
Hematopoietic stem cell transplantation
Young Adult
Fanconi anemia
hemic and lymphatic diseases
Internal medicine
medicine
Humans
Child
Preparative Regimen
Chemotherapy
Hematology
business.industry
Hematopoietic Stem Cell Transplantation
Bone marrow failure
General Medicine
medicine.disease
Fludarabine
Fanconi Anemia
Treatment Outcome
Child, Preschool
Immunology
Savior sibling
Female
Unrelated Donors
business
medicine.drug
Subjects
Details
- ISSN :
- 14320584 and 09395555
- Volume :
- 94
- Database :
- OpenAIRE
- Journal :
- Annals of Hematology
- Accession number :
- edsair.doi.dedup.....160a4943aa9770c18ff648f811009327