1.
T-cell Depleted Peripheral Blood versus Unmanipulated Bone Marrow in Matched Sibling Transplantation for Aplastic Anemia
Chorão, P., Montoro, J., Balaguer-Roselló, A., Guerreiro, M., Villalba, M., Facal, A., Solves, P., Gómez-Segui, I., Pasquini, M. C., Granados, P., et al
Transplantation and cellular therapy. 2023
Abstract
BACKGROUND Bone marrow (BM) is the recommended stem cell source for hematopoietic stem cell transplantation (HSCT) from matched sibling donor (MSD) in severe aplastic anemia (SAA) for its superior survival and lower graft-versus-host disease (GVHD) outcomes compared to unmanipulated peripheral blood (PB). Nevertheless, studies comparing BM with ex vivo T-cell depleted (TCD) PB have not been reported so far. OBJECTIVES The aim of this study was to compare the transplant outcomes of MSD-HSCT in SAA using PB (with partial ex vivo TCD targeted cell dose grafts) with those using unmanipulated BM. STUDY DESIGN We performed a matched-pair analysis of MSD-HSCT using TCD PB in a single institution with unmanipulated BM transplants in the United States between 2013 and 2019 who were reported to CIBMTR. RESULTS We compared 23 recipients of TCD PB HSCT for SAA (cases) to 69 recipients of unmanipulated BM grafts (controls) who were matched for age, Karnofsky performance status, HSCT-specific comorbidity, time from diagnosis to transplant and recipient CMV serostatus. Here, we found a significantly faster neutrophil and platelet recovery in the TCD PB cohort (P < 0.001 and P = 0.03, respectively), as well as a lower incidence of grade II-IV acute GVHD (0% versus 17%; P = 0.05) and similar overall survival (96% vs 97% at 3 years; P = 0.8). CONCLUSIONS Our study shows that TCD PB can be considered as a safe source for MSD-HSCT in SAA, with potential advantages in engraftment and GVHD that could challenge the standard with BM. These findings provide further insights for future research in a prospective controlled clinical trial.
2.
Hematopoietic Cell Transplantation with Cryopreserved Grafts for Severe Aplastic Anemia
Eapen, M., Zhang, M. J., Tang, X. Y., Lee, S. J., Fei, M. W., Wang, H. L., Hebert, K. M., Arora, M., Chhabra, S., Devine, S. M., et al
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2020
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Abstract
With the COVID-19 pandemic and the ensuing barriers to the collection and transport of donor cells, it is often necessary to collect and cryopreserve grafts before initiation of transplantation conditioning. The effect on transplantation outcomes in nonmalignant disease is unknown. This analysis examined the effect of cryopreservation of related and unrelated donor grafts for transplantation for severe aplastic anemia in the United States during 2013 to 2019. Included are 52 recipients of cryopreserved grafts who were matched for age, donor type, and graft type to 194 recipients who received noncryopreserved grafts. Marginal Cox regression models were built to study the effect of cryopreservation and other risk factors associated with outcomes. We recorded higher 1-year rates of graft failure (hazard ratio [HR], 2.26; 95% confidence interval, 1.17 to 4.35; P=.01) and of 1-year overall mortality (HR, 3.13; 95% CI, 1.60 to 6.11; P=.0008) after transplantation of cryopreserved compared with noncryopreserved grafts, with adjustment for sex, performance score, comorbidity, cytomegalovirus serostatus, and ABO blood group match. The incidence of acute and chronic graft-versus-host disease did not differ between the 2 groups. Adjusted probabilities of 1-year survival were 73% (95% CI, 60% to 84%) in the cryopreserved graft group and 91% (95% CI, 86% to 94%) in the noncryopreserved graft group. These data support the use of noncryopreserved grafts whenever possible in patients with severe aplastic anemia.
PICO Summary
Population
Patients with severe aplastic anaemia, who underwent HSCT and were reported to CIBMTR registry
Intervention
Cryopreseverved graft (n=52)
Comparison
Matched controls who received non-cryopreserved grafts (n=194)
Outcome
. We recorded higher 1-year rates of graft failure and of 1-year overall after transplantation of cryopreserved compared with noncryopreserved grafts, with adjustment for sex, performance score, comorbidity, cytomegalovirus serostatus, and ABO blood group match. The incidence of acute and chronic graft-versus-host disease did not differ between the 2 groups. Adjusted probabilities of 1-year survival were 73% in the cryopreserved graft group and 91% in the noncryopreserved graft group.