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Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Postinduction Treatment for Specific ALL Subgroups

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Treatment options for infants withoutMLLtranslocations

The optimal treatment for infants without MLL translocations also remains unclear.

  1. On the Interfant-99 trial, patients without MLL translocations achieved a relatively favorable outcome with the cytarabine-intensive treatment regimen (4-year EFS was 74%).[13]
  2. A favorable outcome for this subset of patients was obtained in a Japanese study using therapy comparable to that used to treat older children with ALL;[16] however, that study was limited by small numbers (n = 22) and a highly unusual gender distribution (91% males).

Treatment options under clinical evaluation for infants with ALL

Treatment options under clinical evaluation include the following:

  1. Interfant-06 Study Group trial (DCOG-INTERFANT-06) (Different Therapies in Treating Infants With Newly Diagnosed Acute Leukemia): The Interfant-06 Study Group is conducting an international collaborative randomized trial (including sites in the United States) to test whether an ALL/acute myeloid leukemia hybrid regimen might improve outcomes for infants with MLL-rearranged ALL. The role of allogeneic transplantation in first remission is also being assessed in high-risk patients (defined as infants with MLL-rearranged ALL, younger than 6 months, and WBC >300,000 /µL) or poor peripheral blood response to steroid prophase. Infants with MLL-rearranged ALL with high MRD at end of consolidation phase are also eligible for allogeneic HSCT in first remission regardless of other presenting features.
  2. COG-AALL0631(Combination Chemotherapy With or Without Lestaurtinib in Treating Infants With Newly Diagnosed ALL): In this COG study of infant ALL, an FLT3 inhibitor, lestaurtinib, is being studied in infants with MLL rearrangement. Infants with MLL rearrangement are known to have a high level of FLT3 mRNA expression and lestaurtinib has been shown to selectively kill MLL-rearranged ALL cells in vitro and in vivo.[22] This study combines lestaurtinib with intensive chemotherapy previously utilized in POG-P9407. An initial safety/activity phase has been completed and the efficacy phase in which children will be randomly assigned to chemotherapy with or without lestaurtinib is now underway. Infants with germline MLL are nonrandomly assigned to less-intensive chemotherapy without lestaurtinib.

Adolescents and Young Adults With ALL

Adolescents and young adults with ALL have been recognized as high risk for decades. Outcomes in almost all studies of treatment are inferior in this age group compared with children younger than 10 years.[23,24,25] The reasons for this difference include more frequent presentation of adverse prognostic factors at diagnosis, including the following:

  • T-cell immunophenotype.
  • Philadelphia chromosome–positivity (Ph+).
  • Lower incidence of favorable cytogenetic abnormalities.
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