Not to be missed

Don't miss out on the Editors pick of key recently published articles

Ruxolitinib in corticosteroid-refractory graft-versus-host disease after allogeneic stem cell transplantation: a multicenter survey
Despite major improvements in allogeneic hematopoietic cell transplantation over past decades, corticosteroid-refractory GVHD remains a clinical challenge and causes high mortality. Preclinical evidence and pilot data indicate anti-inflammatory properties of the JAK1/2 inhibitor ruxolitinib. In order to provide clinical evidence prior to maturation of clinical trial data, this study reports outcome data from 95 patients who had received ruxolitinib as salvage therapy for steroid refractory GVHD. Efficacy was very promising with an overall response rate of 81.5% in case of acute GVHD and 85.4% in case of chronic GVHD. Cytopenia and cytomegalovirus-reactivation were observed during ruxolitinib treatment. Ruxolitinib may constitute a promising new treatment option for steroid refractory GVHD that should be validated in a prospective trial.

Defining and treating high-risk multiple myeloma
Multiple myeloma is more recently being recognized as a heterogeneous group of disease with variability in outcomes based on specific clinical and biologic predictors. MM patients can be categorized into standard, intermediate and high risk for disease relapse, morbidity and mortality. With this expert review, the authors summarize diagnostic and therapeutic options in high risk myeloma. High-risk features include old age, poor performance status, comorbidities, primary plasma cell leukemia, extramedullary disease, deletion 17p, t(4;14) and high-risk gene expression profiling signatures.

Comparison of minimal residual disease as outcome predictor for AML patients in first complete remission undergoing myeloablative or nonmyeloablative allogeneic hematopoietic cell transplantation
Measurable residual disease (MRD), sometimes incorrectly termed minimal residual disease, is a hot topic in leukaemia-therapy. Although MRD-testing is of widely-accepted value in persons with ALL, the value in AML is less certain. Persons with AML in remission who are MRD-test-positive clearly have a greater likelihood of relapse than those who are MRD-test-negative. However, both results are associated with high rates of false-positives and -negatives which limits applying results of MRD-testing to subject-level therapy decisions. This report shows the adverse impact of a positive MRD-test on leukemia relapse risk in persons with AML receiving conventional allotransplants also operates after less intensive conditioning transplants.

Protracted dormancy of pre-leukemic stem cells and A tale of two siblings: two cases of AML arising from a single pre-leukemic DNMT3A mutant clone
Sometimes we think we know more about how leukemia develops than we really do. Two recent reports show our limitations. Ford and colleagues present data from extensive immune and genomic analyses of a child with ALL who developed AML two decades later. Both leukemias arose from the same dormant leukemia stem cell. The evolutionary advance of such dormancy is clear. The report by Hahn and colleagues has parallels. Transplantation of a leukemia stem cell (perhaps pre-leukemia stem cell is a better term) gave rise to AML in sibling. The founder mutation was maintained but subsequent driver mutations were discordant. Conclusions from these reports have important implications regarding our knowledge of how leukemia develops, what is remission and how to interpret results of testing for MRD.