New treatments for severe blood disorders in bone marrow

When people get myelodysplastic neoplasms (MDS), it affects the normal development of blood cells. Severe cases, called higher-risk MDS, progress quickly, cause painful symptoms, and may turn into acute leukemia. Patients who can’t undergo intense treatments like stem cell transplants or high-dose chemotherapy have limited options. Professor Uwe Platzbecker, from the University of Leipzig Medical Center, led an international trial testing a new treatment combination. The results are published in ‘The Lancet’ journal.

About 4,000 people in Germany are diagnosed with myelodysplastic neoplasms (MDS) each year. MDS can cause anemia, infections, and an increased risk of bleeding. Higher-risk MDS patients have a lower life expectancy. They are often treated with demanding methods like stem cell transplantation or chemotherapy. However, those who can’t undergo these treatments have limited options.

In an international trial at 54 sites across 17 countries, researchers tested a new treatment combination involving hypomethylating agents (HMA) and sabatolimab, an immunotherapeutic agent. Sabatolimab targets specific areas of immune and tumor cells, belonging to a class known as immune checkpoint inhibitors.

This trial, led by Professor Uwe Platzbecker from Leipzig University, is the first randomized study exploring immunotherapy in combination with the standard of care for myeloid neoplasms, providing hope for new approaches in higher-risk MDS.

In this study, adding sabatolimab to the standard treatment didn’t significantly improve response rates or progression-free survival.

However, sabatolimab showed a manageable safety profile, with common side effects like reduced white blood cells and platelets. Despite these findings, researchers still see potential. They are conducting a phase 3 trial – the final stage of drug development – to explore the overall survival benefit of combining sabatolimab with azacitidine, a commonly used treatment. Sponsored by Novartis, this study aims to provide more insights into treatment options for patients with limited choices.

Severe blood disorders in the bone marrow, particularly higher-risk myelodysplastic neoplasms (MDS), did not yield significant improvements in response rates or progression-free survival with the addition of sabatolimab. Despite this, the manageable safety profile observed and the potential seen by researchers have paved the way for a follow-up randomized phase 3 trial.

Sponsored by Novartis, this ongoing study investigates the overall survival benefit of combining sabatolimab with azacitidine, offering hope for further advancements in addressing the limited treatment options for individuals with severe bone marrow blood disorders.

Journal reference:

Amer M Zeidan, Amer M Zeidan et al., Sabatolimab plus hypomethylating agents in previously untreated patients with higher-risk myelodysplastic syndromes (STIMULUS-MDS1): a randomised, double-blind, placebo-controlled, phase 2 trial. The Lancet. DOI: 10.1016/S2352-3026(23)00333-2.

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