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Allogeneic transplant fails to confer survival benefit for pediatric leukemia subtype
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Allogeneic hematopoietic stem cell transplantation did not significantly improve survival compared with chemotherapy alone among children with newly diagnosed hypodiploid acute lymphoblastic leukemia, according to results of a retrospective study published in Journal of Clinical Oncology.
However, treatment guided by minimal residual disease (MRD) levels appeared associated with favorable outcomes, as did high hypodiploidity with 44 chromosomes and MRD-negative status after remission induction, results showed.
“With the availability of several new therapies, current patients [with hypodiploid ALL] should have better prognosis,” Ching-Hon Pui, MD, chair of the department of oncology at St. Jude Children’s Research Hospital, told HemOnc Today. “It is reasonable to conduct studies to test if the addition of blinatumomab (Blincyto, Amgen), inotuzumab (Besponsa, Pfizer Oncology) or venetoclax (Venclexta; AbbVie, Genentech) along with intensive chemotherapy will improve outcomes of [these] patients. It’s an effort that could be effective for patients with positive MRD at a low level, or even those with negative MRD at the end of remission induction. If the use of chimeric antigen receptor [CAR] T-cell therapy will improve outcomes of individuals with high MRD at the end of induction, oncologists can reserve transplantation for salvage therapy.”
The analysis included 272 children (median age, 9.8 years; range, 0.6-19.5; 151 male) with hypodiploid ALL who were enrolled in 16 cooperative study groups between 1997 and 2013.
Researchers analyzed clinical and biological characteristics, early therapeutic responses determined by MRD, treatment with or without MRD-stratified protocols, and allogeneic HSCT to determine their impact on outcomes.
Four patients died during remission induction.
Median follow-up was 6.6 years.
Results showed a 5-year EFS rate of 55.1% (95% CI, 49.3-61.5) and a 5-year OS rate of 61.2% (95% CI, 55.5-67.4).
However, researchers observed higher EFS rates among patients with negative MRD at the end of remission induction (75%; 95% CI, 66-85), high hypodiploidy with 44 chromosomes (74%; 95% CI, 61-89) and treatment in MRD-stratified protocols (62%; 95% CI, 55-69).
Further analysis, after excluding patients with high hypodiploidy and adjusting for transplantation wait time and covariables, showed 5-year DFS of 59% (95% CI, 46.5-75) among the 42 patients who underwent allogeneic HSCT and 51.5% (95% CI, 44.7-59.4) among the 186 patients who received only chemotherapy. The difference was not statistically significant, Pui noted, especially considering the long-term risks of radiation related to transplantation.
The study’s lack of randomization and retrospective nature served as limitations.
“It is possible to treat patients with CAR T-cell therapy in a research setting because hypodiploid ALL is rare and the number of patients [with hypodiploid ALL] with high MRD is quite small,” Pui said. “CAR T-cell therapy is expensive — around $500 million using commercial products — but it is less costly than the costs associated with allogeneic transplantation at most centers.” – by John DeRosier
For more information:
Ching-Hon Pui, MD, can be reached at St. Jude Children’s Research Hospital, MS 260, Room C6056, 262 Danny Thomas Place, Memphis, TN 38105.
Disclosures: NCI funded this study. Pui reports honoraria from Amgen and Bristol-Myers Squibb; a consultant/advisory role with Novartis; and travel, accommodations and expenses from Amgen and Sanofi. Please see the study for all other authors’ relevant financial disclosures.
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