Bone marrow transplant using fludarabine-based reduced intensity conditioning regimen with in vivo T cell depletion in patients with Fanconi anemia.
Lev GorfinkelCarolyn DemskyFarzana PashankarGary KupferNiketa C ShahPublished in: Pediatric transplantation (2021)
FA is the most common cause of inherited BMF syndromes. The only cure for BMF in FA remains HSCT. Due to DNA instability in FA, RIC has been used to decrease immediate and late complications of HSCT. Most FA conditioning regimens in mismatched and unrelated donor transplants rely on TBI, which increases the risk of secondary malignancies. Most of the non-TBI conditioning regimens use an ex vivo T-cell depletion approach, but this is not feasible at all pediatric stem cell transplant programs. To evaluate the success of HSCT in patients with FA using non-TBI conditioning regimens with in vivo T-cell depletion approach. HSCT using non-TBI based conditioning was performed on two siblings with FA. The first sibling underwent matched unrelated donor transplant with a BM graft using fludarabine, alemtuzumab, busulfan, and cyclophosphamide conditioning and cyclosporine and mycophenolate as GVHD prophylaxis. The second sibling underwent MSD transplant with UCB and BM grafts using similar approach, but without busulfan and mycophenolate. Both siblings had engraftment without signs of acute or chronic GVHD. Acute post-transplant complications included brief viral reactivations. At last follow-up, both siblings continued to have full immune reconstitution with stable chimerism. Conditioning regimens without radiation and inclusion of alemtuzumab can lead to successful engraftment without development of GVHD and reduce risk of developing secondary neoplasms, even with unrelated donor transplants.
Keyphrases
- traumatic brain injury
- allogeneic hematopoietic stem cell transplantation
- hematopoietic stem cell
- bone marrow
- stem cells
- liver failure
- severe traumatic brain injury
- sars cov
- mild traumatic brain injury
- low dose
- drug induced
- respiratory failure
- acute myeloid leukemia
- mesenchymal stem cells
- chronic kidney disease
- acute lymphoblastic leukemia
- aortic dissection
- risk factors
- high intensity
- high dose
- extracorporeal membrane oxygenation
- acute respiratory distress syndrome
- autism spectrum disorder
- circulating tumor cells