Acute Myeloid Leukemia in Infants: Biology and Treatment

Abstract

Children aged 0–2 years (i.e., infants) with acute myeloid leukemia (AML) are a peculiar subgroup of patients in the childhood AML scenario. They present with distinctive biological and clinical characteristics, including a high prevalence of prognostically unfavorable risk factors and an increased susceptibility to therapy-related toxicity. Remarkable improvements have been achieved over the last two decades in the treatment of these patients and their outcome is becoming superimposable to that of the older age groups. In this review, we will focus on peculiarities of this young subgroup of children with AML, describing their clinical presentation, the biology of disease, and factors influencing outcome. Treatment results and toxicity data reported by major collaborative groups are also summarized and compared.

Acute Myeloid Leukemia in Pediatric Patients: A Review About Current Diagnostic and Treatment Approaches

“…This allows for the destruction of leukemia cells with the hope of avoiding side effects or as little late side effects as possible. With regards to infant AML, the most powerful prognostic factor for the outcome has been found to be favorable cytogenetics and a blast count of less than 5% after induction therapy [49].…”

Acute myeloid leukemia in children. Prospects for the optimization of treatment (review)

“…У детей до 2 лет обнаружены специфические трансло-кации, которые пока не внесены в классификатор ВОЗ. К ним относятся t(7;12)(q36;p13)/HLZB9-MNX1; t(11;12)(p15;q13)/NUP98-KDM5A и t(1;22)(p13;q13)/ RBM15-MKL1 [11]. В частности, транслокация t(1;12) (p15;q13) ассоциирована с М7-подтипом ОМЛ, трансло-кация (7;12)(q36;p13) обычно сопровождается трисо-мией 19-й хромосомы или реже трисомией 8-й хромо-сомы.…”

“…Когда было доказано, что различные MLL-гибридные бел-ки образуют совокупность с дизраптором теломер-ной передачи сигнала, метилтрансферазой DOT1L, появилась возможность использовать ингибитор DOTIL-EPZ004777, что послужило основанием для таргетироания DOT1L при лейкозе с MLL-r. В насто-ящее время ведется исследование (ClinicalTrials.gov -NCT01684150) эффективности этого вида эпигенети-ческого таргетирования [11].…”

Factors Influencing the Outcome of Hematopoietic Stem Cell Transplantation in Pediatric Acute Myeloid Leukemia: Single Institution Results from Saudi Arabia

“…Geographic variation, ethnicity, initial response to treatment and molecular and cytogenetic aberrations affect the outcome [3,4] and in some studies, mitochondrial genome alterations have been found with an increased incidence of high risk AML in children [5]. Similarly, abnormalities of chromosome 5 & 7, presence of FLT3/ITD and MRD by flow cytometry, t (6; 9) (p23; q34), MLL gene rearrangement and infancy all point to higher risk AML patients [6][7][8][9]. Despite some promising results in the last few years, there are still 35% patients die of the disease and the survivors are left with long term complications [10].…”