Treatment Approaches for Therapy-Related AML, MDS Based on Mutation Profiles

Reviewing Real-World Use of Beti-Cel in Transfusion-Dependent β-Thalassemia

Phase 2 data support the potential of revumenib to advance the standard of care for patients with relapsed/refractory NPM1-mutated acute myeloid leukemia.

Therapies like betibeglogene autotemcel have been “life-changing” for patients with β-thalassemia, according to Nora M. Gibson, MD, MSCE.

Nora M. Gibson, MD, MSCE;Taha Al-Juhaishi, MD

May 5th 2025

Bone Marrow Test Reveals Improved Survival in NPM1 and FLT3-Mutated AML

Therapies such as betibeglogene autotemcel have been “life-changing” for patients with β-thalassemia, according to Nora M. Gibson, MD, MSCE.

Tim Cortese

April 30th 2025

Leveraging Ex Vivo Gene Therapy Advancements in Hemoglobinopathies and Metabolic Diseases

This study conducted within the phase 3 AML17 and AML19 trials revealed an OS rate of 69% in patients monitored for MRD and 58% in those not monitored.

A panel of expert pharmacists provides their perspectives on gene therapy agents such as exagamglogene autotemcel in the management of sickle cell disease.

Alexis K. Kuhn, PharmD, BCOP ;Katie Bruce, PharmD, BCPPS;Susie Long, PharmD

October 21st 2024

Concurrent Statin Use With Treatment Improves Survival in CLL/SLL

A panel of expert pharmacists provides their perspectives on gene therapy agents such as exagamglogene autotemcel in the management of sickle cell disease.

Univariate and multivariate analyses showed no significant association between statin use and grade 3 or higher toxicities in patients with CLL or SLL.

FDA Rejects Breakthrough Designation, Approval for Itolizumab in Acute GVHD

Univariate and multivariate analyses showed no significant association between statin use and grade 3 or higher toxicities in patients with CLL or SLL.

Based on FDA feedback, the developers plan to discontinue the phase 3 EQUATOR study, which evaluated itolizumab in acute graft-versus-host disease.

Revumenib Yields Meaningful Benefit in NPM1-Mutated Acute Myeloid Leukemia

Based on FDA feedback, the developers plan to discontinue the phase 3 EQUATOR study, which evaluated itolizumab in acute graft-versus-host disease.

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Russ Conroy

May 9th 2025

Reviewing Real-World Use of Beti-Cel in Transfusion-Dependent β-Thalassemia

Phase 2 data support the potential of revumenib to advance the standard of care for patients with relapsed/refractory NPM1-mutated acute myeloid leukemia.

Nora M. Gibson, MD, MSCE;Taha Al-Juhaishi, MD

May 5th 2025

Bone Marrow Test Reveals Improved Survival in NPM1 and FLT3-Mutated AML

Therapies such as betibeglogene autotemcel have been “life-changing” for patients with β-thalassemia, according to Nora M. Gibson, MD, MSCE.

Tim Cortese

April 30th 2025

Leveraging Ex Vivo Gene Therapy Advancements in Hemoglobinopathies and Metabolic Diseases

This study conducted within the phase 3 AML17 and AML19 trials revealed an OS rate of 69% in patients monitored for MRD and 58% in those not monitored.

Alexis K. Kuhn, PharmD, BCOP ;Katie Bruce, PharmD, BCPPS;Susie Long, PharmD

October 21st 2024

Concurrent Statin Use With Treatment Improves Survival in CLL/SLL

A panel of expert pharmacists provides their perspectives on gene therapy agents such as exagamglogene autotemcel in the management of sickle cell disease.

FDA Rejects Breakthrough Designation, Approval for Itolizumab in Acute GVHD

Univariate and multivariate analyses showed no significant association between statin use and grade 3 or higher toxicities in patients with CLL or SLL.