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Use of Post-Transplant Cyclophosphamide with Nonmyeloablative Conditioning in HLA-Haploidentical Stem Cell Transplants for Sickle Cell Disease

10 August 2019


Background: Hematopoietic stem cell transplantation is the only curative therapy for individuals with sickle cell disease but due to the limited donor pool and complications with adult infusions, new methods are in development for applicability to all populations. The new method utilizes a treatment regimen developed by John Hopkins with only small trials currently being performed.

Methods: A comprehensive search on the most up to date information was performed with MEDLINE, PubMed, MEDLINE-Ovid, EBSCO-Host including all databases, and Web of Science with the terms cyclophosphamide or cytoxan, bone marrow transplant(ation) or stem cell, and sickle cell. Articles and abstracts generated were screened and analyzed by one independent reviewer via inclusion and exclusion criteria and related publication appraised in PubMed. Studies were assessed for quality using GRADE criteria.

Results: Five studies using HLA-haploidentical patient-donor related matches with nonmyeloablative techniques have shown promise in decreasing toxicity to patients while providing an alternative, curative treatment for individuals who previously did not have that option. Nonmyeloablation via John Hopkins protocol with post-transplant cyclophosphamide (PTCy) show minimal acute/chronic graft-versus-host disease effects similar to what is seen in HLA-matched sibling donors but with decreased stable engraftment.

Conclusion: A combination of peripheral blood stem cell (PBSC) and bone marrow transfusions in haploidentical patients from all trials show stable engraftment with minimal cGVHD. Further studies to increase efficacy are needed to modify this conditioning regimen with a need for a large randomized control trial with increased TBI and Thiotepa needed to support findings presented.


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