An analysis of the success of therapy for children and young people with hypodiploid acute lymphoblastic leukaemia (ALL) has found that allogenic stem cell transplantation provides no substantial survival benefit.
However, patients receiving risk-directed therapy, which involves adjusting dose intensity by examining markers of minimal residual disease (MRD), was associated with better patient outcomes.
Researchers at St. Jude Children's Research Hospital, Memphis, Tennessee, USA, published their analysis in the Journal of Clinical Oncology.
Hypodiploid ALL is a high-risk form of the disease which involves leukaemic cells with 25 to 44 chromosomes instead of the normal 46. This patient group are generally treated with intensive chemotherapy, followed by allogeneic bone marrow transplantation, which brings associated risks.
In the retrospective study, which analysed the outcomes of 306 patients from 16 different trials, 58% of patients overall became long-term survivors. However, the researchers found in subgroup analyses that five-year event-free survival was 75% for the patients who were MRD-negative after initial chemotherapy, and 74% for those with ‘high hypodiploidy’ with 44 chromosomes.
Allogenic transplantation did not significantly improve overall survival, compared to chemotherapy alone.
Lead researcher, Dr Ching-Hon Pui, said: "This study confirms our earlier observation that patients with hypodiploid acute lymphoblastic leukaemia who have no evidence of minimal residual disease after remission induction therapy should not be transplanted.
"We recommend continued treatment with intensive chemotherapy."
The researchers say it remains unclear how to improve outcomes for MRD-positive patients with fewer than 44 chromosomes after remission induction chemotherapy.
Source: Pui, C.-H., Rebora. P., Schrappe, M., Attarbaschi, A., Baruchel, A., Basso, G., Cavé, H., Elitzur, S., Koh, K., Liu, H.-C., Paulsson, K., Pieters, R., Silverman, L.B., Stary, J., Vora, A., Yeoh, A., Harrison, C.J., Valsecchi, M.G., on behalf of the Ponte di Legno Childhood ALL Working Group. (2019) “Outcome of Children With Hypodiploid Acute Lymphoblastic Leukemia: A Retrospective Multinational Study”, Journal of Clinical Oncology, available at doi: 10.1200/JCO.18.00822
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