Guideline for treating relapsed or refractory myeloid leukemia in children with Down syndrome

被引：1

作者：

Miladinovic, Milica ^{[1
]}

Reinhardt, Dirk ^{[2
]}

Hasle, Henrik ^{[3
]}

Goemans, Bianca F. ^{[4
]}

Tomizawa, Daisuke ^{[5
]}

Hitzler, Johann ^{[6
]}

Klusmann, Jan-Henning ^{[1
]}

机构：

[1] Goethe Univ Frankfurt, Dept Pediat, Theodor Stern Kai 7, D-60596 Frankfurt, Germany

[2] Univ Hosp Essen, Pediat Hematol & Oncol, Pediat 3, Essen, Germany

[3] Aarhus Univ Hosp Skejby, Dept Pediat, Aarhus, Denmark

[4] Princess Maxima Ctr Pediat Oncol, Utrecht, Netherlands

[5] Natl Ctr Child Hlth & Dev, Childrens Canc Ctr, Div Leukemia & Lymphoma, Tokyo, Japan

[6] Univ Toronto, Hosp Sick Children, Div Hematol Oncol, Toronto, ON, Canada

来源：

PEDIATRIC BLOOD & CANCER | 2024年 / 71卷 / 09期

关键词：

acute myeloid leukemia; Down syndrome; guideline; trisomy; 21; ACUTE MYELOGENOUS LEUKEMIA; TRANSIENT MYELOPROLIFERATIVE DISORDER; BONE-MARROW-TRANSPLANTATION; STEM-CELL TRANSPLANTATION; NATURAL-HISTORY; MEGAKARYOBLASTIC LEUKEMIA; VENOOCCLUSIVE DISEASE; GATA1; MUTATIONS; RISK; AML;

D O I：

10.1002/pbc.31141

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Treatment of relapsed and refractory myeloid leukemia in Down syndrome (r/r ML-DS) poses significant challenges, as prognosis is dire and there is no established standard treatment. This guideline provides treatment recommendations based on a literature review and collection of expert opinions, aiming to improve overall and event-free survival of patients. Treatment options include fludarabine and cytarabine (FLA) +/- gemtuzumab ozogamicin (GO), azacytidine (AZA) +/- panobinostat, and hematopoietic stem cell transplantation (HSCT). Preferred approaches are AZA +/- panobinostat for cases with low blast count or FLA +/- GO for cases with high blast count, followed by HSCT after remission. Further research is crucial for the investigation of targeted therapies (e.g., BH3 mimetics, LSD1, JAK inhibitors).

引用

页数：9

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