Hematology With Afaf E. Osman, MD

Oncology / Hematology

Dr. Osman is an assistant professor at the University of Utah Huntsman Cancer Institute where she treats myeloid malignancies. Her research interest is in myeloid precursor conditions including clonal hematopoiesis.

Progress in Low-Risk MDS: Latest Developments

Dear readers,

This version of my MDS newsletter is focused on low-risk MDS. Much of the recent development in the field of MDS has been in improving the anemia of patients with lower-risk MDS. Not too long ago, these patients had very limited options consisting of long-term transfusion therapies and erythropoiesis-stimulating agents (ESAs). Today, the landscape of available treatments for patients with lower-risk MDS is vastly different.

Firstly, we start with the newest FDA approval for the treatment of anemia in this patient group: Imetelstat. This is an exciting development as Imetelstat represents a first-in-class drug approval. The mechanism of action is very interesting, with Imetelstat inhibiting the telomerase enzyme, which the malignant clone relies on to support its lengthy telomeres.

Secondly, the EHA meeting just concluded in Madrid. and updated data from COMMANDS was presented. This showed continued impressive results for luspatercept in comparison to ESAs in the first-line setting for patients with lower-risk MDS. The median treatment duration of 130 weeks for luspatercept is certainly promising.

The next article discusses an important aspect in the treatment of transfusion dependent patients with lower risk MDS: iron chelation therapy. Iron chelation was deemed controversial in the past, but there is certainly a group of patients with lower risk MDS and iron overload who benefit from it. It’s a relatively benign intervention that should be individually considered in these patients. The article is part of the excellent “How I Treat” series published in Blood.

Finally, more data is emerging on thrombosis in VEXAS. The ability to detect UBA1 mutations is being rapidly added to MDS molecular panels. I assume that clinicians will diagnose more VEXAS cases partially due to the improvement in molecular testing. In this paper, both arterial and venous thrombotic events were observed in patients with VEXAS. The rate of venous thrombosis is about 40%, a significant number that calls for close monitoring of these patients for thrombotic events. A significant subset of patients with VEXAS also have concomitant MDS (mostly low risk MDS).

I hope this edition of my newsletter helped shed some light on the arising issues in lower risk MDS!

Best Regards,

Afaf Osman, MD