Bone Marrow Transplantation最新文献

Belumosudil is a first in class ROCK2-inhibitor approved by the FDA for the 3rd line treatment of chronic graft-versus-host disease (cGvHD). In this retrospective real-world analysis, we report safety and efficacy data of belumosudil treatment from 5 German/Swiss transplant centers. A total of 33 adult patients (median age 59 years) with moderate (n = 2) or severe (n = 31) cGvHD were treated on individual request due to lack of EMA approval. The patient cohort had a long history of cGvHD (median 44 months) and was heavily pretreated (median 4 prior lines). The overall response rate was 42% (95%CI, 25-60%) including organ responses in all organs except the liver (n = 2). The median time to response was 3 months (range, 1-9 months) and 8 of 14 patients (57%) had a durable response at last follow-up. One-third of patients had at least a 50% reduction in concomitant corticosteroid dosage. Median failure-free survival and median overall survival were 16.5 and 23.1 months, respectively. Adverse events ≥CTCAE grade 3 were reported in 27% of patients, with a predominance of infectious events, including one fatal course. The results are consistent with previous prospective trials including a favorable safety profile, while acknowledging the challenges of a heavily pretreated patient cohort.

Graft-versus-host disease (GvHD) is one of the most common and troublesome complications after allogeneic hematopoietic stem cell transplantation (HSCT). Despite adequate GvHD prophylaxis, 30-50% of the patients still develop acute or chronic GvHD, often requiring multiple lines of therapy. Therefore, it is crucial to closely monitor the onset and the response of GvHD to therapies to identify the best available treatment for each patient. Currently, some applications (desktop or mobile) that allow to score GvHD severity at the bedside are available. However, none of the published systems is designed to record ongoing therapies and to upload data in a database, which can support both the clinical decision-making process as well as data collection. To this aim, we developed two Shiny apps: aGvHDtrackR for acute GvHD and cGvHDtrackR for chronic GvHD. These applications record GvHD grading alongside the therapies used for each patient and allow to export of the data in a longitudinal patient-specific database. This is of help for the clinical management of patients and for future multicentric studies on GvHD.

In this real-world study, 153 adult T-cell lymphoblastic lymphoma (T-LBL) patients from sixteen centers in Shanghai were enrolled. Out of them, 103 (67.3%) achieved complete remission (CR). The 2-year overall survival (OS) and progression-free survival (PFS) were 56.3% and 47.6%, respectively. In multivariate analysis, CR after induction treatment significantly improved the OS (p = 0.002) and PFS (p = 0.001). Among CR patients, allogeneic peripheral blood stem cell transplantation (allo-PBSCT) significantly lowered the cumulative incidence of relapse (CIR) compared to autologous PBSCT (p = 0.043) and non-SCT (p = 0.001). Among patients undergoing allo-PBSCT in CR, early (within four induction courses) and late CR (after four induction courses) didn't impact the prognosis with similar 2-year OS (p = 0.590), PFS (p = 0.858), CIR (p = 0.50), and non-relapse mortality (NRM) (p = 0.110). Early and deferred allo-PBSCT for early CR patients also yielded similar 2-year OS (p = 0.640), PFS (p = 0.970), CIR (p = 0.994), and NRM (p = 0.974). As a time-dependent covariate, allo-PBSCT presented a positive effect on PFS (p = 0.018) and had a trend toward better OS (p = 0.064). These data suggested that allo-PBSCT should be considered for T-LBL patients upon achieving CR to enhance survival and reduce relapse risk.

The treatment of relapsed/refractory acute myeloid leukemia (AML) is associated with a dismal prognosis. The allogeneic hematopoietic cell transplantation (allo-HCT) is frequently performed as salvage therapy. Reduced intensity conditioning protocols have been developed with the aim of reducing the leukemia burden without increasing their toxicity. We compared the reduced intensity conditioning FM140 (fludarabine, 150 mg/m²; melphalan 140 mg/m²) with FBM110 (fludarabine 150 mg/m²; BCNU, also known as carmustine, 300-400 mg/m²; and melphalan 110 mg/m²). From the European Bone Marrow Transplantation (EBMT) Acute Leukemia Working Party registry, we identified 293 adult patients (FM140, n = 118 and FBM110, n = 175) with AML with relapsed/refractory disease prior to allo-HCT. There were some differences such as age (FM140 = 59.5 years vs. FBM110 = 65.1 years, p < 0.001) and graft-versus-host disease (GvHD) prophylaxis based on in vivo T-cell depletion (TCD, FM140 = 39% vs. FBM110 = 75%, p < 0.001). No differences were observed between FM140- and FBM110-treated patients regarding overall survival (OS) (2-year OS: 39.3% vs. 45.7%, p = 0.58), progression-free survival (PFS) (2-year PFS: 36.1% vs. 37.3%, p = 0.69), non-relapse mortality (NRM) (2-year NRM: 15.3% vs. 25.7%, p = 0.10) and relapse incidence (RI) (2-year RI: 48.6% vs. 37.0%, p = 0.7). In conclusion, despite differences in age and GvHD prophylaxis, AML patients with active disease undergoing allo-HCT after FBM110 conditioning showed similar outcomes compared to FM140.