Treatment of children with newly diagnosed acute promyelocytic leukemia with arsenic trioxide: a single center experience

Abstract

A total of 11 children (five males and six females) with hypergranular type of acute promyelocytic leukemia (APML) were treated with intravenous arsenic trioxide (As 2 O 3 ) between December 1998 and October 2003. Eight cycles of As 2 O 3 (0.15 mg/kg/day) were administered (induction, consolidation and six cycles of maintenance) over a period of 12 months. The median WBC count at diagnosis was 3400/mm 3 (range: 800-9800). In all, 10 patients (91%) achieved hematological remission at a mean duration of 48 days (range: 41-60) with all 10 patients achieving molecular remission at a median duration of 81 days (range: 64-109). Toxicity was minimal with leukocytosis in six patients, ichthyosis and hyperpigmentation of skin in five and mild peripheral neuropathy in one patient. One patient who relapsed 6 months after completing therapy achieved a second hematological and molecular remission with As 2 O 3 . With a median follow-up of 30 months (range: 4-62), the overall (OS) survival is 91% with a relapse-free survival (RFS) of 81%. As 2 O 3 achieves hematological and molecular remission in majority of newly diagnosed children with APML with minimal toxicity, but long-term follow-up is required to evaluate late effects of As 2 O 3 and study the minimum dose and duration required for a sustained remission.

Treatment strategy and results in children treated on three Dutch Childhood Oncology Group acute myeloid leukemia trials

“…The differential treatment strategies for children with acute promyelocytic leukemia 40 and AML in DS 41 are clear examples of this approach. In addition, the role of new targeted drugs, such as gemtuzumab ozogamicin and FLT3 inhibitors, needs to be established by future clinical studies.…”

How to manage acute promyelocytic leukemia

“…67 Recent literature supports our data in that the CR rate and EFS were relatively higher in the working groups using ATO/ATRA combination 68,69 compared with the groups using ATO alone. 59,60,62 GIMEMA group carried out a randomized phase III study to compare ATO þ ATRA versus standard ATRA þ anthracycline-based chemotherapy (AIDA regimen) for newly diagnosed non high-risk APL. We are eagerly anticipating their results.…”

Pediatric Acute Myeloid Leukemia

“…(Mathews, et al 2010) This has also been piloted in 11 children, with similar encouraging findings. (George, et al 2004) When this is confirmed in larger studies treatment of APL without chemotherapy may be feasible, especially when no long-term toxicities from arsenic treatment emerge.…”