Reduced Intensity Vs. Standard Conditioning Followed By Allogeneic Stem Cell Transplantation for Patients with MDS or Secondary AML: A Prospective, Randomized Phase III Study of the Chronic Malignancies Working Party of the EBMT (RICMAC-Trial)

• Published in: Blood 2014-12, Vol.124 (21), p.320-320
• Main Authors: Kröger, Nicolaus, Brand, Ronald, Niederwieser, Dietger, Platzbecker, Uwe, Hübel, Kai, Weber, Thomas, Robin, Marie, Stelljes, Matthias, Afanasiev, Boris V, Heim, Dominik, Lambertenghi Deliliers, Giorgio, Onida, Francesco, Dreger, Peter, Pini, Massimo, Guidi, Stefano, Volin, Liisa, Gramatzki, Martin, Bethge, Wolfgang A., Poire, Xavier, Kobbe, Guido, van Os, Marleen, Iacobelli, Simona, de Witte, Theo
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V124.21.320.320

▪ Introduction Retrospective studies in MDS/sAML suggest that reducing the intensity of the conditioning regimen prior to allogeneic stem cell transplantation reduces the risk of non-relapse mortality but is associated with a higher risk of relapse, but prospective randomized studies for MDS are lacking so far. Patients and Methods Within the Chronic Malignancies Working Party (CMWP) of EBMT, we performed a prospective randomized trial comparing a busulfan based (Busulfan 8mg/kg orally or equivalent dosis intravenously (iv) plus fludarabin 180mg/m²) reduced intensity conditioning regimen (RIC) and a standard myeloablative busulfan (Busulfan 16mg/kg orally or equivalent dosis iv plus cyclophosphamide 120mg/kg) based regimen (MAC) in patients with MDS or sAML ( than 5%. The primary endpoint of the study was 1 year non-relapse mortality.The median age of the patients was 51.4 years (r.19-64y). Donors were HLA-identical sibling (n=34), matched unrelated (n=59) and mismatched related or unrelated (n=30). The patients were well distributed in both arms regarding age, gender, IPSS risk profile, number of blasts at transplantation, donor source and mismatch donor. Results Leukocyte count more than 1.0 x 10e9/L and platelet count more than 50x10e9/L at day 28 was reached in 90 % and 70% after RIC and in 92% and 77% after MAC, respectively Acute GvHD II-IV was noted in 29% after RIC and 32% after MAC. Chronic GvHD was seen in 61% after RIC and 64% after MAC. The cumulative incidence (CI) of non-relapse mortality (NRM) after 1 year was 17% (95% CI 8-26%) after RIC and 28% (95% CI 16-39%) after MAC (p=0.18). The CI of NRM at 1 year after HLA-identical sibling transplantation was lower than after unrelated transplantation after RIC (0% vs 23%, p=0.06) as well after MAC (17% vs 32%; p=0.18) The CI of relapse at 2 years was 18% (95% CI 8-27%) after RIC and 15% (95% CI 5-24%) after MAC (p=0.5), resulting in a 2 year relapse-free and overall survival of 61% (95% CI 48-73%) and 74% (95%