Myelofibrosis treatment
Ten years ago, if you had asked a specialist hematologist about the treatment of myelofibrosis, you would have received a sad face and a multitude of medications that do not directly deal with the disease. Myelofibrosis is a genetic problem with manifestations at a cellular level, and unless the treatment tackles the genetic problem, it will persist and progress. Targeted genetic therapy greatly improves the outcome of myelofibrosis and all myeloproliferative neoplasms.
Treatment is usually tailored according to the severity of the condition as well as the presence of complications. Some complications such as the enlargement of the spleen may need surgical treatment especially if it causes hypersplenism. To understand how treatment works, we have to deal with 2 main problems, the genetic one and the one related to the symptoms. Both approaches are done at the same time.
1) Ruxolitinib
Ruxolitinib is a chemotherapeutic agent that belongs to a class called JAK inhibitiors. JAK stand for Janus associated kinases and such enzymes play a central role in the development of myelofibrosis. However, the mutation that the drug deals with is only found in half the cases of myelofibrosis. Another sibling to Ruxolitinib is fedratinib, and it has shown great promise in initial trials, which is why it has been recently recommended for some advanced cases. It has to be understood though that those 2 drugs have not yet proved an effect like that of tyrosine kinase inhibitor drugs like imatinib or nilotinib on chronic leukemias, but they are the first step towards a specific treatment for myelofibrosis.