AP-HP Paris France

Oct 19, 2024 | movementdisorders.onlinelibrary.wiley.com | Emmanuel Roze |Institut du Cerveau |AP-HP Paris France

The Ultragenyx@-sponsored phase 3 randomized controlled study evaluating the effect of triheptanoin to treat paroxysmal episodes of movement disorders related to Glut1 deficiency syndrome (Glut1-DS) failed to demonstrate efficacy.1 This was unexpected considering the rationale supporting the use of triheptanoin,2 and the striking results of preliminary investigator-driven open-label studies that showed over 90% reduction of paroxysmal movement disorders in patients with Glut1-DS,3, 4 a...

Oct 19, 2024 | onlinelibrary.wiley.com | Hôpital Saint-Antoine |AP-HP Paris France |Antoine Capes

Within the past decade, chimeric antigen receptor (CAR)-T-cell therapy has been established as a key treatment for relapsed/refractory B-cell malignancies.1 However, this breakthrough in terms of efficacy came with some specific toxicities such as cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS).2, 3 In addition to CRS and ICANS, cytopenias are frequent after CAR-T-cell infusions. Rejeski et al.

Jul 23, 2023 | onlinelibrary.wiley.com | Florent Malard |Hôpital Saint-Antoine |AP-HP Paris France

Abstract Chronic graft-versus-host disease (cGvHD) remains the most important long-term complication of allogeneic hematopoietic cell transplantation (allo-HCT), but the field has seen significant changes in the last decade.