Survival Outcomes of Children with Relapsed or Refractory Myeloid Leukemia Associated with Down syndrome
العنوان: | Survival Outcomes of Children with Relapsed or Refractory Myeloid Leukemia Associated with Down syndrome |
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المؤلفون: | Nikhil Raghuram, Kentaro Nakashima, Syaza Ab Rahman, Evangelia Antoniou, Torjus Skajaa, Pietro Merli, Anupam Verma, Karen R. Rabin, Catherine Aftandilian, Rishi Sury Kotecha, Daniel Ka Leung Cheuk, Kirsi Jahnukainen, Alexandra Kolenova, Walentyna Balwierz, Alice Norton, Maureen M O'Brien, Sonia Cellot, Ashley Chopek, Nira Arad-Cohen, Bianca F. Goemans, Marta Rojas-Vasquez, Hany Ariffin, Jack Bartram, Edward A Kolb, Franco Locatelli, Daisuke Hasegawa, Jan-Henning Klusmann, Henrik Hasle, Bryan McGuire, Lillian Sung, Johann K. Hitzler |
المصدر: | Raghuram, N, Nakashima, K, Ab Rahman, S, Antoniou, E, Skajaa, T, Merli, P, Verma, A, Rabin, K R, Aftandilian, C, Kotecha, R S, Cheuk, D K L, Jahnukainen, K, Kolenova, A, Balwierz, W, Norton, A, O'Brien, M M, Cellot, S, Chopek, A, Arad-Cohen, N, Goemans, B F, Rojas-Vasquez, M, Ariffin, H, Bartram, J, Kolb, E A, Locatelli, F, Hasegawa, D, Klusmann, J-H, Hasle, H, McGuire, B, Sung, L & Hitzler, J K 2023, ' Survival Outcomes of Children with Relapsed or Refractory Myeloid Leukemia Associated with Down syndrome ', Blood Advances . https://doi.org/10.1182/bloodadvances.2022009381 |
بيانات النشر: | American Society of Hematology, 2023. |
سنة النشر: | 2023 |
مصطلحات موضوعية: | Hematology |
الوصف: | 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 2000-2021. Median time from diagnosis to relapse was 6.8 (range 1.1 - 45.5) months. Three-year event-free (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) (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 CR prior to 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 due to 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. |
تدمد: | 2473-9537 2473-9529 |
URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::31a0fcd1b97bcceef6f43bd5f4ef5a6f https://doi.org/10.1182/bloodadvances.2022009381 |
حقوق: | OPEN |
رقم الأكسشن: | edsair.doi.dedup.....31a0fcd1b97bcceef6f43bd5f4ef5a6f |
قاعدة البيانات: | OpenAIRE |
تدمد: | 24739537 24739529 |
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