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Pyrimethamine in the myeloproliferative disorders: a forgotten treatment?
Abstract:
Eight patients with myeloproliferative disorders, five with polycythaemia rubra vera (PRV) and three with essential thrombocythaemia (ET), have been treated with the anti-folate drug Pyrimethamine for periods ranging from 1 to 24 years. In PRV this treatment was comparable in efficacy to that achieved with Busulphan or radioactive phosphorus, but required more frequent supervision. One patient was controlled on Pyrimethamine, having failed on conventional treatment. The major side effect was thrombocytopenia which was rapidly reversible on stopping the drug. In ET, Pyrimethamine produced satisfactory control of the platelet count and thrombocytopenia did not arise. No neurological sequelae were encountered. One patient developed a non-Hodgkin's lymphoma of the gut, but there were no other cases of secondary malignancy. Pyrimethamine may still have a role in the treatment of selected cases of myeloproliferative disorders.
Insights
Pyrimethamine effectively treated myeloproliferative disorders like polycythaemia rubra vera (PRV) and essential thrombocythaemia (ET). This anti-folate drug showed comparable efficacy to standard treatments, with manageable side effects like reversible thrombocytopenia.
Area of Science:
- Hematology
- Pharmacology
Background:
- Myeloproliferative disorders (MPDs) encompass conditions like polycythaemia rubra vera (PRV) and essential thrombocythaemia (ET).
- Conventional treatments for MPDs include Busulphan and radioactive phosphorus, often requiring careful management.
Observation:
- Eight patients with MPDs (5 PRV, 3 ET) received Pyrimethamine, an anti-folate drug, for 1-24 years.
- Treatment efficacy was assessed by disease control and adverse events.
- Patient outcomes and side effects were monitored throughout the treatment period.
Findings:
- Pyrimethamine demonstrated efficacy comparable to Busulphan or radioactive phosphorus in PRV patients, though requiring more frequent supervision.
- One PRV patient achieved disease control with Pyrimethamine after failing conventional therapies.
- Pyrimethamine effectively controlled platelet counts in ET patients without inducing thrombocytopenia. The primary side effect observed was rapidly reversible thrombocytopenia, with no neurological sequelae or secondary malignancies reported in most cases.
Implications:
- Pyrimethamine may serve as a valuable therapeutic option for selected patients with myeloproliferative disorders.
- The drug's efficacy and manageable side effect profile warrant consideration in treatment-resistant cases.
- Further research into Pyrimethamine's role in MPD management is suggested.