Whether Momelotinib requires long-term use and long-term safety assessment

Momelotinib is a JAK inhibitor used to treat intermediate- and high-risk myelofibrosis. Its treatment mechanism determines that most patients often need to take it long-term or even continuously. Because myelofibrosis is a chronic progressive disease, symptoms (such as splenomegaly, anemia, general weakness) usually worsen again within weeks to months after drug withdrawal. Therefore, the clinical goal is to maintain long-term stability rather than short-term cure. Discontinuation or dose adjustment will only be considered when obvious adverse reactions occur, the efficacy is weakened, or the treatment regimen needs to be changed.

In terms of long-term efficacy, research shows that molotinib has the unique advantage of improving anemia, allowing some patients to reduce their dependence on blood transfusions, and can continuously control spleen volume and disease-related symptoms. Compared with other JAK inhibitors that tend to aggravate anemia, molotinib improves erythropoiesis by inhibiting the ACVR1 pathway, so the benefits of long-term treatment are relatively stable. Most patients can maintain symptom relief after continuous use for several months. Stopping the drug will often lead to a rebound of symptoms. Therefore, long-term treatment under the advice of a doctor can better ensure the stability of the disease.

From the perspective of long-term safety, molotinib is generally well tolerated. Common side effects include mild to moderate diarrhea, nausea, headache or fatigue, most of which can be gradually relieved with continued medication or improved through symptomatic treatment. No obvious cumulative toxicity has been found in long-term observational studies, and there is no high risk of severe infection or significant immunosuppression. However, it is still necessary to regularly monitor changes in blood routine, liver and kidney function, and systemic symptoms, and to deal with intolerance in a timely manner. For patients whose anemia has significantly improved, the safety benefits of long-term use are more prominent.

In general, molotinib needs to be taken for a long time in most cases and is a maintenance treatment drug rather than a staged course of treatment. During long-term treatment, patients need to work closely with their doctors to regularly evaluate efficacy, monitor side effects, and adjust dosage according to individual responses to ensure maximum clinical benefit and safety. If there is a decrease in efficacy or serious adverse reactions, it is necessary to promptly evaluate whether to switch to other JAK inhibitors or accept hematopoietic stem cell transplantation and other alternatives.

Reference materials:https://www.drugs.com/