DECEASED DONOR BONE MARROW TRANSPLANT: FIRST EVER HUMAN CASE REPORT of SUCCESSFUL ENGRAFTMENT after INFUSION of A CRYOPRESERVED DECEASED DONOR BONE MARROW in A PATIENT with AML

Document Type

Conference Proceeding

Publication Date

6-12-2025

Publication Title

HemaSphere

Keywords

budesonide, cyclophosphamide, fludarabine, melphalan, mycophenolate mofetil, tacrolimus, acute graft versus host disease, acute myeloid leukemia, adult, African American, aged, blood donor, bone marrow, bone marrow transplantation, case report, chimera, clinical article, conference abstract, deceased donor, drug combination, drug therapy, engraftment, female, graft rejection, graft versus host reaction, hematopoietic stem cell, HLA matching, human, human tissue, infant, Karnofsky Performance Status, living donor, microchimerism, mismatched unrelated donor, neutrophil, neutrophil count, organ donor, platelet count, reduced intensity conditioning, special situation for pharmacovigilance, therapy, vertebra body

Abstract

Background: Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative therapy for a variety of hematologic diseases. Clinical data now confirm that T-cells contained within the donor graft exert a "graft-versus-malignancy" effect. Use of HLA-mismatched unrelated donors (MMUD) would greatly increase treatment options for all patients, but particularly for those who are ethnically diverse. OSSIUM cryopreserved hematopoietic progenitor cell (HFC) marrow from deceased organ donors provides an "on-demand" HFC graft for patients without available living donors. Here we report the first human to be treated with unrelated, minimally manipulated allogeneic cryopreserved HPC marrow for hematopoietic reconstitution after reduced intensity conditioning. This marrow is processed through proprietary means from vertebral bodies of deceased organ donors. Case Summary:, A 68-year-old African American female patient with acute myeloid leukemia in CR1 was transplanted using cryopreserved HFC marrow from a cadaveric donor at Henry Ford Health, Detroit, MI. Prior to transplant the patient received fludarabine 30 mg/m2 (Day-6 to Day-2), melphalan 140 mg/m2 ( Day-6) and TBI 2Gy in one fraction, as part of reduced intensity conditioning regimen. The transplant was approved by the local 1KB. Methods High resolution HLA matching between donor and recipient was 4/8. All organ function were reported to be in normal range and KPS score was 80%. Patient received 5.27x10" cryopreserved CD34+ cells/kg. The infusion lasted for approximately 76 minutes. The HPC, marrow product infusion was uneventful. Graft-versus-host disease (GVHD) prophylaxis consisted of tacrolimus, mycophenolate mofetil and two doses of post-transplant cyclophosphamide (PTCy) at a dose of 50 mg/kg administered on Days +3 and +4. Results Neutrophil engraftment was reported on Day +16 (neutrophil count D+16-570, D+17-1500, D+18-3690 cells/L) and platelet engraftment was reported on Day+ 21 (platelet count Day + 21-20,000, D+22-22,000, D+23-34,000 cells/L) post-transplant. Full blood donor chimerism was reported on Day +30. Bone marrow on Day +60 showed 100% donor chimerism. Based on clinical suspicion, on Day +54 acute upper GI GVHD (Grade II, stage 1) was diagnosed. The acute GVHD is being treated with budesonide 3 mg by mouth daily and tacrolimus 2.5 mg twice daily, and to date her GVHD is well controlled. Summary/Conclusion The successful engraftment in this case demonstrates the potential of cryopreserved HPC marrow from mismatched deceased organ donors as a viable option for HCT. HCT with a MMUD cryopreserved HPC marrow from a deceased organ donor can play a significant role in expanding access to HCT for patients who have unmet needs, and it can be made available "on demand". Clinical studies are currently being conducted to further evaluate the safely and effectiveness of a cryopreserved HPC marrow product.

Volume

9

Issue

S1

First Page

309

Last Page

310

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