Juvenile myelomonocytic leukemia medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]
Overview
There is no internationally accepted treatment protocol for JMML. Currently, 2 clinical treatment protocols most widely used to study JMML and improve treatment for these children are geographically-based:
- North America: The Children’s Oncology Group (COG) JMML Study
- Europe: The European Working Group for Myelodysplastic Syndromes (EWOG-MDS) JMML Study
- Other clinical trials open to patients with JMML may be searched for at the NIH Clinical Trials website.
The following procedures are used in one or both of the current clinical trials listed above:
- Chemotherapy
- Radiotherapy
- Splenectomy
Chemotherapy/Pharmacologic Treatment
The role of chemotherapy against JMML before bone marrow transplant has not been studied and is still unknown. Chemotherapy by itself has proven unable to bring about long-term survival in JMML.
Low-dose conventional chemotherapy
Studies have shown no influence from low-dose conventional chemotherapy on JMML patients’ length of survival. Some combinations of 6-mercaptopurine with other chemotherapy drugs have produced results such as decrease in organ size and increase or normalization of platelet and leukocyte count.
Intensive chemotherapy
Complete remission from JMML has not been possible through use of intensive chemotherapy, but it is still used at times because it has improved the condition of a small but significant number of JMML patients who do not display an aggressive disease. The COG JMML Study administers 2 cycles of fludarabine and cytarabine for 5 consecutive days along with 13-cis retinoic acid during and afterwards. The EWOG-MDS JMML Study, however, does not recommend intensive chemotherapy before bone marrow transplant.
13-cis Retinoic acid (a.k.a. Accutane)
In the lab, 13-cis-retinoic acid has been proven to inhibit the growth of JMML cells. The COG JMML Study therefore includes 13-cis-retinoic acid in its treatment protocol, though its therapeutic value for JMML remains controversial.