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Published on: September 19, 2013
Prednisone response is the strongest predictor of treatment outcome in infant acute lymphoblastic leukemia
M Dördelmann1, A Reiter, A Borkhardt
1Department of Pediatric Hematology and Oncology, Medical School Hannover, Hannover, Germany. The ALL-BFM Group.
Insights
Infant acute lymphoblastic leukemia (ALL) prognosis depends on prednisone response. Poor responders (PPR) have a dismal outlook, necessitating new treatment strategies for better outcomes in infant ALL.
Area of Science:
- Pediatric Oncology
- Hematology
- Clinical Trials
Background:
- Infant acute lymphoblastic leukemia (ALL) presents unique challenges with distinct biological and clinical features.
- Identifying prognostic factors is crucial for tailoring treatment strategies in this vulnerable population.
Purpose of the Study:
- To define prognostic factors in infant ALL by analyzing outcomes based on presenting features and early response to prednisone.
- To evaluate the effectiveness of conventional versus intensified therapy based on prednisone response.
Main Methods:
- Retrospective analysis of 106 infants (age ≤12 months) from 3 Berlin-Frankfurt-Münster (BFM) ALL trials (1983-1990).
- Prednisone response assessed by day 8 blast count after prednisone prephase and intrathecal methotrexate.
- Patients categorized as prednisone good responders (PGR; <1,000 blasts/µL) or poor responders (PPR; ≥1,000 blasts/µL).
Main Results:
- Infant ALL frequently showed high white blood cell counts, CNS leukemia, lack of CD10 expression, and 11q23 rearrangements, all associated with inferior outcomes.
- Event-free survival at 6 years was significantly better for PGR (53% ± 6%) compared to PPR (15% ± 7%).
- Poor prednisone response (PPR) was the strongest adverse prognostic factor (RR 3.3; 95% CI, 1.9-5.8).
Conclusions:
- Prednisone response is a critical prognostic indicator in infant ALL.
- Infants with a good prednisone response (PGR) may benefit from conventional therapy.
- Intensified therapeutic strategies, including early intensification or bone marrow transplantation, are essential for prednisone poor responders (PPR).
Abstract:
To define prognostic factors in infant acute lymphoblastic leukemia (ALL), the outcome of 106 infants (age /=1,000 blasts/microL) received intensified therapy. Infant ALL was characterized by a high incidence of a white blood cell count greater than 100 x 10(3)/microL (57%), central nervous system leukemia (24%), lack of CD10 expression (59%), 11q23 rearrangement (49%) including the translocation t(4;11) (29%), and a comparatively high proportion of PPR (26%), which were all significantly associated with inferior outcome by univariate analysis. The estimated probability for an event-free survival at 6 years (pEFS) was by far better for PGR compared with PPR, who had a dismal prognosis despite intensified treatment (pEFS, 53% +/- 6% v 15% +/- 7%, P =.0001). Infant PGR, who were less than 6 months of age (n = 40), lacked CD10 expression (n = 43), and/or had an 11q23 rearrangement (n = 17) fared significantly better compared with corresponding PPR, as indicated by a pEFS of 44% +/- 8%, 49% +/- 8%, and 41% +/- 12%, respectively. In multivariate analysis, PPR was the strongest adverse prognostic factor (relative risk, 3.3; 95% confidence interval, 1.9 to 5.8; P <.0001). Infants with PGR, comprising a major subgroup (74%) among infants, might successfully be treated with conventional therapy, whereas PPR require new therapeutic strategies, including early treatment intensification or bone marrow transplantation in first remission.
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