Blood Cancer Journal最新文献

Pomalidomide has been shown to improve survival in patients with relapsed/refractory myeloma (RRMM). However, the optimal pomalidomide-based combinations in RRMM are not known. This study compared pomalidomide, cyclophosphamide, dexamethasone (PCD) with pomalidomide and dexamethasone (PD) in Asian patients with RRMM. Patients were randomly assigned to receive PCD or PD. Patients received pomalidomide at 4 mg from days 1 to 21, dexamethasone at 40 mg once a week, and those in the PCD arm received cyclophosphamide at 400 mg once weekly for three weeks. The primary endpoint was progression-free survival. One hundred and twenty-two patients were randomized (62 PCD, 60 PD). Baseline characteristics were comparable between both arms. The median prior lines of therapy were three. At a median follow-up of 13.5 (median range 9-18) months, median progression free survival was significantly longer at 10.9 months (95% confidence interval 7.1-27.7) in the PCD group compared with 5.8 months (95% CI, 4.4-6.9) in the PD group (hazard ratio 0.43; p < 0.001). Adverse events rates were similar in both arms. The most common grade ≥3 adverse events were hematological toxicities and pneumonia. 34 deaths occurred during the study (PCD: 17; PD: 17) and three were deemed to be related to study treatment. In Asian patients with RRMM after exposure to proteasome inhibitor and lenalidomide, progression-free survival was significantly prolonged with the addition of cyclophosphamide to PD, with a manageable safety profile.Trial ID: Registered at www.clinicaltrials.gov : NCT03143049.

Allogeneic stem cell transplant (ASCT) remains the only curative option in chronic myelomonocytic leukemia (CMML). We retrospectively analyzed 138 CMML patients who underwent ASCT at the Mayo Clinic. Patients who transitioned to ASCT while in chronic phase (Group A) displayed superior post-transplant survival (PTS), compared to those in whom ASCT was performed after blast transformation (BT; Group B) (median 95 vs. 16 months; p = 0.01). In Group A, PTS was superior in patients with <5% bone marrow (BM) blasts at time of ASCT (median 164 vs. 13.5 months; p = 0.01). Other predictors of superior PTS included day-100 BM blast <5% or normal cytogenetics (median 164 vs. 18 months; p = 0.01) or presence of chronic graft-versus-host-disease (GVHD; median 164 vs. 26 months; p = 0.01). Pre-ASCT hypomethylating agent exposure (HR = 2.03; p = 0.03), and receiving more than one line of pre-ASCT chemotherapy (p = 0.01) predicted inferior PTS. In multivariable analysis, predictors of superior GVHD-free and relapse-free survival (GRFS) included the use of myeloablative conditioning and the absence of morphologically or cytogenetically apparent disease at day-100. The use of post-transplant cyclophosphamide (PTCy) was associated with a higher cumulative incidence of relapse (p = 0.02) and numerically inferior PTS (p = 0.1). Group B patients also appeared to benefit from achieving BM blast <5% at the time of ASCT (p = 0.4) as well as at day-100 (p = 0.01), in terms of PTS, while full chimerism and normal cytogenetics at day-100 were associated with superior GRFS. These observations support the value of ASCT in CMML, especially if performed prior to BT and in the presence of <5% BM blasts at the time of ASCT. Additionally, the observed detrimental impact of PTCy requires additional studies to confirm and investigate the underlying mechanisms.