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December 8, 2025BloodOpen Access

Optimizing outcomes and accessibility of matched sibling donor transplantation for severe hemoglobinopathies in low-resource settings

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Authors

RARajat Agarwal

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Overview

Observational analysis achieved high thalassemia-free and GVHD-free survival in matched sibling transplants, suggesting accessibility can improve outcomes.

Key Points

  • Overall survival was 95.6%, with thalassemia-free survival at 93.9%, indicating excellent outcomes from the transplantation strategy.
  • Utilizing a cost-effective regimen, this approach involved fludarabine and dexa, achieving promising results for severe hemoglobinopathies.
  • The cohort included 113 patients, with a median age of 8.1 years, demonstrating disease management in a pediatric population.
  • High transplant-related mortality of 4.4% was noted, but mitigated by effective supportive care for complications.

Cite This Study

Rajat Agarwal (2025) studied this question.

synapsesocial.com/papers/69362f5a4fa91c937236dba6https://doi.org/10.1182/blood-2025-5987
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion dependent thalassemia in LMICs2026
  2. 2Analysis of efficacy and prognostic factors in transfusion-dependent thalassemia following allogeneic hematopoietic stem cell transplantation using the ‘gx-07-tm’ regimen2025
  3. 3Encouraging Outcomes of Hematopoeitic Stem Cell Transplantation in Pediatric Sickle Cell Disease- A Decade-Long Experience from the Developing World2025
  4. 4Hematopoietic stem cell transplantation with reduced toxicity myeloablative conditioning regimen for children and adults with sickle cell disease: A brazilian single center experience.2025
  5. 5Reduced intensity haploidentical bone marrow transplantation in children with severe sickle cell disease (SCD): BMT CTN 15072025 · 4 citations