Stem cell transplantation to treat patients with a serious but very rare form of chronic blood cancer called juvenile myelomonocytic leukemia (JMML) improves the condition, finds a study.
Allogeneic hematopoietic stem cell transplantation (HSCT) involves the transplantation of stem cells from a donor, which may be derived from bone marrow, peripheral blood or umbilical cord blood.
The recipient's immune system is usually destroyed with radiation or chemotherapy before the transplantation.
"The lack of transplant-related mortality in the group of children we studied at the Children's Center for Cancer and Blood Diseases suggests that BUMEL (Intravenous Busulfan and Melphalan) may represent a successful (HSCT) high-dose chemotherapy regimen," said lead author Hisham Abdel-Azim from the Children's Hospital Los Angeles.
"It is also possible that administering conventional dose chemotherapy, before HSCT, to patients with more progressive disease may have contributed to the improved outcomes," Abdel-Azim said in the journal Blood.
The study looked at children with JMML who underwent HSCT at Children's Hospital Los Angeles.
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All of the patients were alive and in clinical remission.
It is the only reported cure for JMML; however best outcomes of the therapy have shown that only half the patients can be cured from this disease.
There is currently no standard conditioning regimen for children with JMML undergoing HSCT.