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• W2023334285 abstract "UCBT offers a cure for thalassemia and CB has advantages as a stem cell source for minorities, where thalassemia is prevalent. Previously mortality risks outweigh benefits and cell dose is critical for UCBT. With strategies that maximize cell dose - using non-red cell reduced plasma depleted (PD) CB, no post-thaw wash (NW), and double cord transplantation (DCT), promising results may be achieved with UCBT in selected thalassemics. Between 7/01 and 9/06, 47 UCBT were performed after myeloablative therapy in 41 pediatric patients with thalassemia major (7 DCT & 1 re-transplant) at 14 transplant centers (TC) using 81% PD CB with 91% NW. Patient status: 19 Pesaro class 1, 4 class 2, 1 class 3, and 17 status unknown. The median age was 5 yo (range 0.3-20 yo) with a median weight of 18 kg (range 8-45 kg). The data was audited by external agencies. No significant adverse events were observed despite major ABO incompatibility in some cases. Kaplan-Meier estimates of ANC500, platelet 20K and platelet 50K engraftment rate with donor chimerism were 86±6%, 74±8% and 76±8%, and median times to ANC500, platelet 20K and platelet 50K engraftment were 16 days (range 11-32), 38 days (range 16-99) and 57 days (range 30-106) after transplantation respectively. 7 patients died with 2 deaths prior to day 20 and one traumatic head injury. 32 patients are known to be alive, no survival data for 2 patients, 5 with autologous recovery, and 34 engrafted with a mean and median follow up time of 440 and 243 days respectively (range 8-1,750 days) as of 9/18/2006. Median day to hospital discharge was day +64 (range 22-121 days). Experienced TC (>5 cases of UCBT for thalassemia) were compared with less experienced TC, and CB that were post-thaw washed were compared with CB that were NW. Results appear to show improvement with TC experience and NW. The role of plasma depletion vs. RBC depletion was studied by matching age, weight, TNC dose, #HLA matches, conditioning regimen, TC experience, and NW status between the two groups , and results trended towards improvements in engraftment and DFS for PD CB. To our knowledge, this is the largest multi-institutional UCBT series for thalassemia and demonstrate that UCBT may be a promising approach for the curative therapy of thalassemia major and engraftment of 93±6% and 1-year DFS of 88±8% are attainable with experience and judicious patient selection when cell dose is optimized with PD CBU, no post-thaw wash and DBT when necessary." @default.
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• W2023334285 date "2007-02-01" @default.
• W2023334285 modified "2023-09-26" @default.
• W2023334285 title "166: Unrelated cord blood transplantation (UCBT) for transfusion-dependent thalassemia – a retrospective audited analysis of 41 consecutive patients" @default.
• W2023334285 doi "https://doi.org/10.1016/j.bbmt.2006.12.170" @default.
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