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Treatment options for infants without KMT2A rearrangements

Source: Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: April 21, 2025 · Captured 2026-09-09.

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Context: Postinduction Treatment for Specific ALL Subgroups / Infants With ALL

The optimal treatment for infants without KMT2A rearrangements also remains unclear, in part because of the paucity of data on the use of standard ALL regimens used in older children.

On the Interfant-99 trial, patients without KMT2A rearrangements achieved a relatively favorable outcome with the cytarabine-intensive treatment regimen (4-year EFS rate, 74%).[23]

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The COG P9407 (NCT00002756) trial of intensified chemotherapy reported a 5-year EFS rate of 70% in infants without the KMT2A rearrangement.[24][Level of evidence B4]

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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.[27] However, that study was limited by small numbers (n = 22) and a highly unusual sex distribution (91% males).

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On the Interfant-06 study, the 6-year EFS rate for infants without KMT2A rearrangements was 73.9%, and the OS rate was 87.2%.[40]; [30][Level of evidence B4]

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On the COG AALL0631 (NCT00557193) study, infants without KMT2A rearrangements received the same intensified chemotherapy backbone as those with the KMT2A rearrangement.[41]

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On the COG AALL0631 (NCT00557193) study, infants without KMT2A rearrangements received the same intensified chemotherapy backbone as those with the KMT2A rearrangement.[41]

The 5-year EFS rate was 87.3% (± 4.7%), and the 5-year OS rate was 93.6% (± 3.5%) for 64 infants without KMT2A rearrangements.

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Preserved source evidence · Not medical advice