HLA-Haploidentical Transplantation in Children with Primary Immunodeficiency Using Post Cyclophosphamide
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION(2020)
摘要
Introduction Hematopoietic Stem Cell Transplantation (HCT) is considered the curative treatment for patients with Primary Immune Deficiencies (PIDs). Finding a full matched Human leukocyte donor remains an obstacle for performing the bone marrow transplant in a timely fashion. Haplo-identical HCT is an alternative treatment option for patients, who do not have access to full-matched donor. We are reporting preliminary data on our experience of HCT in PID patients using haplo-identical donor with post-transplant Cyclophosphamide. Methods Eleven transplant naive PID patients were infused from June 2017 to August 2018 using a haplo-identical HLA donor at our institution. Eight (72.7%) of the recipients were male. Median age at infusion was 0.9 years (Min: 5.5 months Max: 2.3 years). Primary indications for transplantation included Severe Combined Immune Deficiency (SCID) in seven (63.6%), Omenn Syndrome in two (18.2%), and Chediak-Higashi syndrome and Combined Immunedeficiency in one (9.1%) each. All received GVHD prophylaxis comprising of Cyclophosphamide, Mycophenolate mofetil (MMF), and Cyclosporine regimen. Conditioning regimen was BU/FLU/Thiotepa (±ATG) in six (54.5%) recipients followed by BU/FLU/ATG in four (36.4%). Source of stem cells was bone marrow in ten patients (90.9%) while for the remaining one child PBSC was utilized. Results Eight patients (72.7%) had ANC engraftment with a median time to ANC recovery 15.5 days (Minimum: 13, maximum: 22) whereas, seven (63.6%) of our patients achieved platelets recovery (Median: 33 days, minimum: 16, maximum: 150). In terms of chimeric studies, all but one patient, engrafted at Day +100. Cumulative incidence of aGVHD was 27.3% (n=3); no chronic GVHD was recorded. At a median follow-up of 18.8 months (95% CI: 14.6-23.1), cumulative probability of overall survival at one year from infusion, for our patients is 72.7% (±13.4%). Hemorrhagic cystitis was seen in only one (9.1%) patient while two (18.2%) experienced mucositis (Grade1 or 2). Three (27.3%) contracted CMV infection and were successfully treated.At one-year follow-up lymphoid chimerism remains u003e95% donor type and CD3:1717 mm3 and CD19 521 mm3 Conclusion Preliminary outcome data on our small cohort of immunodeficiency patients who underwent HCT using Haplo-identical donor with Post-Cy is quite encouraging with durable and stable donor chimerism and CD3 and CD19 engraftment.
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