Early viral reactivation despite excellent immune reconstitution following haploidentical Bone marrow transplant with post‐transplant cytoxan for sickle cell disease

• DOI: 10.1111/tid.13222
• Publication Date: 2019-11-29T16:53:40Z
• Authors: Dilan A. Patel; Nathalie Dhedin; Heidi Chen; Leena Karnik; Katie Gatwood; Katie Culos; Sanjay Mohan; Brian G. Engelhardt; Carrie Kitko; Jim Connelly; Gowri Satyanarayana; Madan Jagasia; Josu De La Fuente; Adetola Kassim
• Keywords: Adult; Transplantation Conditioning; Adolescent; Graft vs Host Disease; Anemia, Sickle Cell; Disease-Free Survival; 3. Good health; Young Adult; 03 medical and health sciences; Clinical Trials, Phase II as Topic; Immune Reconstitution; 0302 clinical medicine; Transplantation, Haploidentical; Humans; Virus Activation; Prospective Studies; Child; Cyclophosphamide; Immunosuppressive Agents; Bone Marrow Transplantation

Haploidentical bone marrow transplant (haplo-BMT) offers near universal donor availability as a curative modality for individuals with severe sickle cell disease (SCD). However, the required intense immunodepletion is associated increased infectious complications. A paucity of data exists on immune reconstitution following haplo-BMT SCD.A multi-institution learning collaborative was developed in context phase II clinical trial non-myeloablative, related post-transplant cyclophosphamide SCD. We report results from cohort 23 patients whom up to one year were available.Median age 14.8 years. Out 23, 18 participants received pre-conditioning azathioprine, hydroxyurea, and hypertransfusions. 70% (16/23) had multiple indications haplo-BMT. observed excellent CD4, CD8, CD19, CD56 cellular subsets by 6 months post transplant. Engraftment rate event-free survival this 100% 96%, respectively. at least viral reactivation or infection, including CMV 35% (8/23), HHV-6 22% (5/23), polyoma virus 17% (4/23), no cases lymphoproliferative disease.Further prospective studies are needed better characterize immunologic basis