Raghuram, Nikhil and Hasegawa, Daisuke and Nakashima, Kentaro and Rahman, Syaza and Antoniou, Evangelia and Skajaa, Torjus and Merli, Pietro and Verma, Anupam and Rabin, Karen R. and Aftandilian, Catherine and Kotecha, Rishi S. and Cheuk, Daniel and Jahnukainen, Kirsi and Kolenova, Alexandra and Balwierz, Walentyna and Norton, Alice and O'Brien, Maureen and Cellot, Sonia and Chopek, Ashley and Arad-Cohen, Nira and Goemans, Bianca and Rojas-Vasquez, Marta and Ariffin, Hany and Bartram, Jack and Kolb, E. Anders and Locatelli, Franco and Klusmann, Jan-Henning and Hasle, Henrik and Mcguire, Bryan and Hasnain, Afia and Sung, Lillian and Hitzler, Johann (2023) Survival outcomes of children with relapsed or refractory myeloid leukemia associated with Down syndrome. Blood Advances, 7 (21). pp. 6532-6539. ISSN 2473-9529, DOI https://doi.org/10.1182/bloodadvances.2022009381.
Full text not available from this repository.Abstract
Children with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population. We conducted a retrospective analysis of international study groups and pediatric oncology centers and identified 62 patients who received treatment with curative intent for r/r ML-DS between year 2000 to 2021. Median time from diagnosis to relapse was 6.8 (range, 1.1-45.5) months. Three-year event-free survival (EFS) and overall survival (OS) were 20.9 +/- 5.3% and 22.1 +/- 5.4%, respectively. Survival was associated with receipt of hematopoietic stem cell transplantation (HSCT) (hazard ratio HR], 0.28), duration of first complete remission (CR1) (HR, 0.31 for > 12 months) and attainment of remission after relapse (HR, 4.03). Patients who achieved complete remission (CR) before HSCT, had an improved OS and EFS of 56.0 +/- 11.8% and 50.5 +/- 11.9%, respectively compared to those who underwent HSCT without CR (3-year OS and EFS of 10.0 +/- 9.5%). Treatment failure after HSCT was predominantly because of disease recurrence (52%) followed by treatment-related mortality (10%). The prognosis of r/r ML-DS remains dismal even in the current treatment period and serve as a reference point for current prognostication and future interventional studies. Clinical trials aimed at improving the survival of patients with r/r ML-DS are needed.
| Item Type: | Article |
|---|---|
| Funders: | International Berlin-Frankfurt-Muenster AML Study Group |
| Subjects: | R Medicine > R Medicine (General) |
| Divisions: | Universiti Malaya Medical Centre (UMMC) |
| Depositing User: | Ms. Juhaida Abd Rahim |
| Date Deposited: | 09 Sep 2025 03:05 |
| Last Modified: | 09 Sep 2025 03:05 |
| URI: | http://eprints.um.edu.my/id/eprint/50600 |
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