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40-Year-Old RA Drug May Be Low-Cost Option for Patients With Myeloproliferative Neoplasms


Patients with polycythemia vera and essential thrombocythemia may be able to reduce their symptoms through a low-cost drug used to treat rheumatoid arthritis (RA), according to a new study in British Journal of Haematology.

Patients with polycythemia vera (PV) and essential thrombocythemia (ET) may be able to reduce their symptoms through a low-cost arthritis drug, according to a new study in British Journal of Haematology.

The authors used methotrexate (MTX) because it is a Janus-associated kinase (JAK)/signal transducers and activators of transcription (STAT) pathway inhibitor, and mutations activating JAK/STAT signaling are key drivers of myeloproliferative neoplasms (MPNs), such as PV and ET.

Patients with PV have an overproduction of red blood cells, and patients with ET have an overproduction of blood-clotting platelets. As a result, they suffer with itching, headaches, weight loss, fatigue, and night sweats. While current treatments for PV and ET control the overproduction issue, they do not address the symptoms that can have a significant impact on quality of life.

“While we still need to undertake a clinical trial to validate these findings, our results are very encouraging and suggest that a simple drug that has been used for nearly 40 years to treat arthritis can provide significant relief to blood cancer sufferers,” Martin Zeidler, BSc, DPhil, Department of Biomedical Science, University of Sheffield.

They identified 11 patients with ET or PV who were already taking low-dose MTX for other diseases. These patients taking MTX had lower symptom scores than patients not on the medication.

In the patients with PV, they reported lower mean scores in 9 of 10 symptoms, and in patients with ET, they reported lower mean scores for all symptoms. The researchers suggested that MTX was suppressing the activity of pro-inflammatory cytokines in the patients, which correlated with the findings that symptoms associated with chronic inflammation were the most reduced.

Current treatments for MPNs not only exclude a large proportion of the population, but also have financial and cost-effectiveness concerns, significant side effects, and loss of efficacy. However, MTX has been used for 40 years and has been shown to be well tolerated with mild or moderate adverse events.

While the findings support the “potential effectiveness” of low-dose MTX in patients with ET and PV, as well as potentially with other MPNs, the authors noted that the data is not conclusive and more studies are needed.

“Given the inflammatory nature of MPN symptoms we suggest that low-dose MTX may represent a low cost, safe and effective treatment for the wider MPN population and propose further studies to test this hypothesis,” the authors wrote.


Francis S, Thomas S, Luben R, et al. Low‐dose methotrexate: potential clinical impact on haematological and constitutional symptoms in myeloproliferative neoplasms [published September 16, 2019]. Br J Haematol. doi: 10.1111/bjh.16193.

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