Ruxolitinib + Fostamatinib for Chronic Graft-versus-Host Disease

This trial explores a new treatment combination for individuals with chronic graft-versus-host disease (cGvHD) who haven't responded well to steroids. Researchers aim to determine a safe and effective dose of fostamatinib (also known as Tavalisse) when combined with ruxolitinib, a standard treatment for cGvHD. Participants should have cGvHD that persists despite steroid use or returns after treatment.

As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new combination therapy.

The trial does not specify if you need to stop taking your current medications, but ongoing systemic therapy for chronic GvHD other than corticosteroids, calcineurin inhibitor, or mycophenolate mofetil is not allowed, except for fewer than 3 weeks of ruxolitinib.

Research has shown that ruxolitinib, one of the drugs tested with fostamatinib in this study, is usually well-tolerated for treating chronic graft-versus-host disease (cGVHD). However, it can cause side effects, with the most common being low blood counts, such as anemia (low red blood cells) and thrombocytopenia (low platelets).

Fostamatinib is also part of this combination, but specific safety information for its use with cGVHD is limited. As this is an early-phase study, the main goal is to find a safe and effective dose. Researchers are still determining how well the treatment is tolerated when both drugs are used together.

Researchers are excited about combining fostamatinib with ruxolitinib for chronic graft-versus-host disease (cGVHD) because it offers a new way to tackle this challenging condition. Most current treatments for cGVHD, like steroids and other immunosuppressants, broadly suppress the immune system. However, fostamatinib targets specific pathways involved in immune responses, potentially reducing side effects and improving efficacy. Additionally, ruxolitinib, already a staple for cGVHD, works by inhibiting different immune pathways. Together, this combination may provide a more tailored approach, potentially leading to better control of symptoms and improved patient outcomes.

Research has shown that ruxolitinib effectively treats chronic graft-versus-host disease (cGVHD), particularly in patients unresponsive to steroids. Studies indicate that patients taking ruxolitinib are three times more likely to improve compared to those receiving the best available therapy. Specifically, ruxolitinib alone demonstrated a 33% improvement rate after six months. In this trial, participants will receive a combination of ruxolitinib and fostamatinib. When used with treatments like ruxolitinib, fostamatinib is thought to enhance effectiveness by targeting specific parts of cGVHD. This combination aims to assist those who don't respond to or rely on steroids.³⁶⁷⁸⁹