Andrew Moreno

DocwireNews

A clinical trial has been conducted on the use of the thrombopoietin receptor agonist eltrombopag in patients with chronic myelomonocytic leukemia (CMML) with severe thrombocytopenia. Its findings were presented in a letter published in Leukemia.
“Carefully escalated eltrombopag induces frequent but transient platelet responses in CMML patients with thrombocytopenia,” the trial investigators wrote.
This open-label, single-arm, phase II trial enrolled 29 patients across 15 centers who had CMML and lower risk disease, with platelets of less than 50 ×109/L and a maximum of 5% bone marrow blasts. The cohort had a median age of 78 years, and 21 patients were male. It had a median platelet count of 29 ×109/L and 12 patients were dependent on platelet transfusions. Twenty-seven patients had undergone centralized genetics, 93% of whom had TET2 mutation, 52% RUNX1 mutation, 37% SRSF2 mutation, 30% ASXL1 mutation, 17% PHF6 mutation, and 15% EZH2 mutation.
Eleven patients in the cohort had undergone at least one prior therapy, seven of whom had received immune thrombocytopenia-directed drugs in the form of steroids or intravenous immunoglobulins.
For at least 12 weeks, patients received eltrombopag at a starting dose of 100 mg once daily, which was later amended to 50 mg/day. Dose was adjustable up to 300 mg/day and patients receiving benefit could continue the treatment for up to 24 months.
Among the reported efficacy results, 48.3% of the cohort achieved a platelet response at 12 weeks and 72.4% had response at any time on the study. The median duration of response in the cohort was 2.6 months, and 34.5% of the cohort had response which lasted six months or longer. The cohort had an overall survival rate of 47% and a two-year progression-free survival rate of 23%.
Regarding safety findings, Grade 3 hepatobiliary adverse events affected 10% of the cohort.
Reference
Rabian F, Chevret S, Gruson B, et al. Eltrombopag in chronic myelomonocytic leukemia with severe thrombocytopenia. A Groupe Francophone des Myélodysplasies (GFM) study. Leukemia. 2024. doi:10.1038/s41375-024-02402-8

Zachary Bessette

FDA Approves Nivolumab and Hyaluronidase-nvhy for Subcutaneous Injection

Colorectal Cancer

Cecilia Brown

Dr. Byers Receives 2025 Edith and Peter O’Donnell Award in Medicine for Discoveries in SCLC

Small Cell Lung Cancer

Lung Cancer Research Foundation® Announces 2024 Scientific Grant Program Awards

Non-Small Cell Lung Cancer

Latest News

New Platelet Response Data: Eltrombopag in CMML With Severe Thrombocytopenia

A phase II trial evaluated use of the thrombopoietin receptor agonist in patients with at least one prior therapy.

About Us

Editorial Policy

Advertising

eNewsletters

Contribute

Contact Us

MashupMD

Blood Cancers Today

Cancer Nursing Today

GU Oncology Now

Urban Health Today

Bile Duct Cancer

Gastroesophageal Cancer

Gastric Cancer

GEP-NETs

GIST

Liver Cancer

Pancreatic Cancer

GI Oncology Now

Anemia

Deep Vein Thrombosis

Essential Thrombocythemia

Hemophilia

Immune Thrombocytopenic Purpura

Myelodysplastic Syndromes

Myelofibrosis

Myeloproliferative Neoplasms

Polycythemia Vera

Pulmonary Embolism

Sickle Cell Disease

Thrombocytopenia

Thrombophilia

Thrombosis

Thrombotic Thrombocytopenic Purpura

Venous Thromboembolism

von Willebrand Disease

Heme Today

Acute Kidney Injury

ADPKD

Anemia and Kidney Disease

Chronic Kidney Disease

COVID-19 and Kidney Disease

Diabetes and Hypertension

End-Stage Renal Disease

Gout

Hyperkalemia

IgAN

Kidney Transplantation

Metabolic Acidosis

Nephrology Times

Mesothelioma

Squamous Cell Lung Carcinoma

Lung Cancers Today

Atherosclerotic Disease

Atrial Fibrillation

Heart Failure

Hypertension

Interventional Cardiology

Lipid Management

Practice Tips With Dr. Lichaa

Preventive Cardiology

Stroke

Cardiology

Breast Cancer

Gynecologic Cancer

Head and Neck Cancer

Skin Cancer

Oncology

Sleep

Urology

Diabetes

Ophthalmology

— Demodex Blepharitis

Neurology

Rheumatology

Gastroenterology

Pulmonology

Infectious Diseases

— HIV

Health and Wellness

Lupus

Future of Medicine

Orthopedics

Pharmacology

Career News

More

Conferences

CardioNerds

The Sleep Source With Dr. Morse

The Oncology Brothers

Rheumatology Rounds With Dr. Maheswaranathan

Healthcare Unfiltered

The Fellow on Call

Partners

Hurricanes & Health Care

Career Center

Expert Interviews

Subscribe