Opinion
Video
Tailoring Therapy: Weighing Evidence, Organ Involvement, and Route of Administration
Panelists discuss how factors such as patient preferences, disease severity, organ-specific involvement, and adverse effect profiles influence the selection of second-line therapies for steroid-refractory chronic graft-vs-host disease (cGVHD) in the context of multiple FDA-approved agents.
Factors Influencing Treatment Selection in Chronic Graft-Vs-Host Disease With Multiple FDA-Approved Agents
As the landscape for treating steroid-refractory chronic graft-vs-host disease (cGVHD) expands with new FDA-approved agents, clinicians face the challenge of choosing the right therapy for each patient. The lack of head-to-head studies means treatment selection is often based on indirect comparisons and multiple patient-specific factors. Here, the panel discusses key elements that influence their decisions, considering the growing availability of treatment options and their distinct profiles.
One major consideration is patient preference, especially with respect to the convenience of treatment. Oral therapies are generally more convenient for patients, offering the option to take medications at home, which can improve adherence and quality of life. On the other hand, intravenous [IV] therapies like ruxolitinib or axatilimab require clinic visits for administration, which may be a deterrent for some patients, especially if they have been post transplant for many years and are reluctant to return for frequent infusions. For patients who struggle with oral intake, such as those with severe esophageal or gastrointestinal [GI] involvement, IV options become an important consideration.
Additionally, clinicians are mindful of the adverse effect profiles of each drug, as ibrutinib has largely fallen out of favor due to its adverse effects, even though it remains a potential option for certain patient groups, especially those with skin and oral involvement. Ruxolitinib, with its favorable evidence, remains a mainstay of therapy, but as with all treatments, clinicians must also be cautious about organ involvement. For example, drugs like ruxolitinib may exacerbate liver dysfunction, making it crucial to weigh the risks and benefits in patients with liver involvement. Real-world data and anecdotal experience further inform these decisions, as clinicians learn more about drug interactions, adverse effects, and efficacy in everyday practice.
In addition to choosing between oral and IV treatments, clinicians must also consider disease severity and organ-specific involvement, adjusting therapy based on whether the patient has severe lung, GI, skin, or liver disease. For instance, in lung involvement, agents like ruxolitinib have shown moderate success, while in severe esophageal or GI cases, drugs with IV formulations may be preferred to avoid complications related to oral intake or absorption.
In this evolving treatment landscape, balancing efficacy, patient preferences, and logistical considerations (such as access to IV treatments and patient ability to adhere to a treatment regimen) will be crucial for optimizing outcomes in patients with cGVHD. While the variety of available therapies provides options, it also requires thoughtful consideration of each patient's unique needs.
