Issue Archive

Goldenberg and colleagues' retrospective analysis of pediatric patients with high-risk, occlusive lower extremity deep vein thrombosis (DVT) suggests that thrombolytic therapy with tPA (with or without mechanical thrombolysis) may decrease the risk of postthrombotic syndrome (PTS).

Boddaert and colleagues show the positive effects of selective iron chelation in young patients with Friedreich ataxia, a devastating inborn error of mitochondrial iron metabolism.

In this issue of Blood, Wagner-Ballon and colleagues provide new insights into the pathogenesis of myelofibrosis with myeloid metaplasia (MMM) and a targeted therapeutic approach of NF-κB inhibition with the proteosome inhibitor bortezomib.

Dok-3 serves as an inhibitory adaptor protein through recruitment of the 5′-inositol phosphatase, SHIP-1. Ng and colleagues have generated a Dok-3–deficient mouse that exhibits hyperresponsive B cells, thereby implicating the adaptor as an important negative regulator of certain signaling events downstream from the antigen receptor.

Chinen and colleagues describe the outcome of gene therapy for 3 young adolescents with XSCID for whom prior attempts at allogeneic bone marrow transplant had failed to achieve significant immunologic reconstitution. Clear-cut clinical benefits have recently been obtained in multiple trials of gene therapy using hematopoietic stem cells (HSCs) for infants with XSCID and adenosine deaminase (ADA)-deficient SCID, and for young adults with chronic granulomatous disease (CGD). Thus, the debate over gene therapy has moved from “will it ever be beneficial?” to “when is it beneficial?” Studies are moving forward exploring the use of gene-corrected autologous HSCs in treating other immune deficiencies (eg, Wiskott-Aldrich syndrome), hemoglobinopathies, leukodystrophies, and mucopolysaccharidoses, as well as multiple applications to oncological diseases.

Mutations in the MYH9 gene give rise to May-Hegglin and related giant-platelet syndromes. In this issue of Blood, Chen and colleagues use mouse models to show that Myh9 acts through the Rho-ROCK pathway as a negative regulator of platelet production in the bone marrow.

In this issue of Blood, Svegliati and colleagues provide new insights into the mechanisms of chronic GVHD development by demonstrating the presence and functional activity of stimulatory anti–PDGF-R autoantibodies in patients with extensive chronic GVHD.