Characteristics and outcomes of children with acute myeloid leukemia and Down syndrome who are ineligible for clinical trials due to severe comorbidities. Issue 11 (18th August 2019)

• Record Type: Journal Article
• Authors: Nakashima, Kentaro; Hasegawa, Daisuke; Tomizawa, Daisuke; Miyamura, Takako; Hama, Asahito; Iwamoto, Shotaro; Terui, Kiminori; Adachi, Souichi; Taga, Takashi
• Is Part Of: Pediatric blood & cancer. Volume 66:Issue 11(2019)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2019-08-18
• Subjects: acute myeloid leukemia -- clinical trial -- comorbidity -- congenital heart disease -- Down syndrome; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.27942
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract: Background: High survival rates of 80‐90% have been reported in recent clinical trials of reduced‐intensity chemotherapies for children with acute myeloid leukemia and Down syndrome (AML‐DS). However, a certain number of children with AML‐DS have complicating comorbidities, including congenital heart disease (CHD), and are therefore ineligible for enrolment in clinical trials. Methods: We retrospectively analyzed the clinical characteristics and outcomes of children with AML‐DS who were excluded from Japanese clinical trials conducted between 2000 and 2015. Results: Twelve children (six males and six females) were identified and were ineligible for CHD ( n = 8) and other comorbidities, including hyperleukocytosis complicated with coagulopathy, severe hemophagocytosis, pulmonary fibrosis, and hypoxic‐ischemic encephalopathy ( n = 1 each). The median age at the diagnosis was 14 months (range, 5 months to 11.5 years). Among all cases, 11 patients were treated with curative intent. Four patients were considered intolerant to intensive chemotherapy and received only low‐dose cytarabine‐based chemotherapy: three failed to achieve remission and died of disease, while one successfully achieved remission but eventually died of infection. Seven cases underwent regular‐intensive chemotherapy for AML‐DS: six were alive and in remission; one had relapsed disease. One patient who received the best supportive care died of disease. Finally, six patients remained in continuous complete remission, while six died. The 5‐year overall survival rate was 51%. Conclusions: The prognosis of AML‐DS patients who received insufficient treatment due to severe complication was poor. The optimal dose intensity of curative chemotherapy for such cases should be explored.