Minimally myelosuppressive regimen for remission induction in pediatric AML: long-term results of an observational study

Abstract

Treatment refusal and death as a result of toxicity account for most treatment failures among children with acute myeloid leukemia (AML) in resource-constrained settings. We recently reported the results of treating children with AML with a combination of low-dose cytarabine and mitoxantrone or omacetaxine mepesuccinate with concurrent granulocyte colony-stimulating factor (G-CSF) (low-dose chemotherapy [LDC]) for remission induction followed by standard postremission strategies. We have now expanded the initial cohort and have provided long-term follow-up. Eighty-three patients with AML were treated with the LDC regimen. During the study period, another 100 children with AML received a standard-dose chemotherapy (SDC) regimen. Complete remission was attained in 88.8% and 86.4% of patients after induction in the LDC and SDC groups, respectively (P = .436). Twenty-two patients in the LDC group received SDC for the second induction course. Significantly more high-risk AML patients were treated with the SDC regimen (P = .035). There were no significant differences between the LDC and SDC groups in 5-year event-free survival (61.4% ± 8.7% vs 65.2% ± 7.4%, respectively; P = .462), overall survival (72.7% ± 6.9% vs 72.5% ± 6.2%, respectively; P = .933), and incidence of relapse (20.5% ± 4.5% vs 17.6% ± 3.9%, respectively; P = .484). Clearance of mutations based on the average variant allele frequency at complete remission in the LDC and SDC groups was 1.9% vs 0.6% (P < .001) after induction I and 0.17% vs 0.078% (P = .052) after induction II. In conclusion, our study corroborated the high remission rate reported for children with AML who received at least 1 course of LDC. The results, although preliminary, also suggest that long-term survival of these children is comparable to that of children who receive SDC regimens.

Pediatric acute myeloid leukemia with t(8;21) and KIT mutation treatment with avapritinib post-stem cell transplantation: a report of four cases

“…They were treated using the CALSIII-AML18 protocol, registered as ChiCTRl800015883 (the registration Qingwei Wang, Yixin Hu, Li Gao and Senlin Zhang contributed equally to this study. date: April 26, 2018), and it is is comparable that the result of long-term survival between standard-dose chemotherapy (SDC) low-dose chemotherapy (LDC) [15]. All four patients underwent HSCT with myeloablative conditioning regimens after achieving complete remission in morphology and the transplantation circumstances were shown in the Table 2.…”

The effect of decitabine-combined minimally myelosuppressive regimen bridged allo-HSCT on the outcomes of pediatric MDS from 10 years’ experience of a single center

“…Then, it has been continuously improved and demonstrated to be efficacious during the past decades mainly among adult MDS/AML population s [13,14]. Intriguingly, pediatric AML patients receiving MMR (one-tenth of standard dose of cytarabine, one-half dose of anthracycline in conjunction with G-CSF) showed similar outcomes and mutation clearance levels, but significantly lower toxicity compared with those receiving standard chemotherapy in our cente r [15,16].…”

“…The AML-type induction chemotherapy was similar to the protocol of AML induction remission therapy used in our cente r [15,16]. The decitabine-combined minimally myelosuppressive regimen (DAC + MMR) included three subtypes of regimens.…”

Homoharringtonine-Based Induction Regimen Improved the Remission Rate and Survival Rate in Chinese Childhood AML: A Report From the CCLG-AML 2015 Protocol Study

“…HHT-based induction regimens were mostly reported in patients with relapsed or refractory AML, chronic myeloid leukemia (CML), or myelodysplastic syndrome. [35][36][37][38][39] Only three studies 14,40,41 evaluated the efficacy of HHT-based chemotherapy in de novo patients, one of which was in patients with adult AML. In one study performed in de novo patients with pediatric AML, 40 a low-dose or standard-dose chemotherapy including HHT achieved a CR of 88.8% or 86.4%, respectively.…”

“… 35 - 39 Only three studies 14 , 40 , 41 evaluated the efficacy of HHT-based chemotherapy in de novo patients, one of which was in patients with adult AML. In one study performed in de novo patients with pediatric AML, 40 a low-dose or standard-dose chemotherapy including HHT achieved a CR of 88.8% or 86.4%, respectively. In another study, 41 72.0% had a CR and the 5-year EFS was 88.0%.…”