An analysis of treatment for a rare form of leukaemia suggests patients derive most benefit from therapies that minimise toxicity, US researchers have reported.
Based at the Children's Hospital Los Angeles, USA, the research team examined findings from 252 studies of mixed-phenotype acute leukaemia (MPAL) over the last 20 years. This research covered 1,499 adult and child patients in 33 countries.
Their metanalysis suggests that starting treatment with "a less-toxic regimen is associated with clear benefits for achieving remission and possibly for long-term survival", they report in the journal Leukemia.
Lead researcher, Dr Etan Orgel, says: "Because this disease is so rare, we haven't had clinical trials with thousands of patients to define the optimal therapy. Instead, we have many small, isolated and often-conflicting case reports published in widely diffuse journals around the world. It's disjointed."
Patients initially treated with therapy usually given for acute lymphoblastic leukaemia were three to five times more likely to achieve complete remission than those treated with therapy usually given for acute myeloid leukaemia. This is the "significantly less toxic" of the two therapies, the researchers say.
"This makes a really convincing case that starting with acute lymphoblastic leukaemia therapy is beneficial on all fronts, from remission to overall survival, if not from increasing survival, then from decreasing side effects," says first author, Dr Maria Maruffi.
Dr Orgel adds: "This research provides key insights to help guide physicians treating patients walking in the door today. But it also highlights the critical need for a clinical trial to definitively determine the best therapy for mixed-phenotype acute leukaemia and help move treatment for this rare disease forward."
Source: Maruffi, M. et al. Leukemia February 27 2018.
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