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Health Status and Health-related Quality of Life Measurement in Pediatric Cancer Clinical Trials

An Examination of the DFCI 00-01 Acute Lymphoblastic Leukemia Protocol

Rae, Charlene, MSc*,†; Furlong, William, MSc*,‡; Feeny, David, PhD*,‡,§; Couchman, Rana, BSc; Silverman, Lewis, MD; Sallan, Stephen, MD; Laverdiere, Caroline, MD#; Clavell, Luis, MD**; Michon, Bruno, MD††; Kelly, Kara, MD‡‡; Larsen, Eric, MD§§; Pullenayegum, Eleanor, PhD∥∥; Athale, Uma, MSc, MD¶¶,##; Barr, Ronald, MBChB, MD¶¶,##

Journal of Pediatric Hematology/Oncology: November 2018 - Volume 40 - Issue 8 - p 580–587
doi: 10.1097/MPH.0000000000001235
Original Articles

Health-related quality of life (HRQL) improved progressively during therapy and beyond in children treated for acute lymphoblastic leukemia on the Dana-Farber Cancer Institute (DFCI) 95-01 protocol. This study aimed to validate that trajectory in a successor study (DFCI 00-01) and to compare the HRQL of patients in the 2 studies. Children aged above 5 years were assessed during each phase of treatment (N=4) and 2 years after completion of therapy. Health status and HRQL were measured using Health Utilities Index (HUI) instruments, HUI2 and HUI3. Quality-adjusted life years (QALYs) were calculated and compared with the general population, and patients treated on DFCI 95-01. Over 5 intervals and 758 HUI assessments, mean HRQL increased progressively from remission induction to the time after treatment (P<0.001). During intensification, high-risk patients had lower HRQL than standard-risk patients (P<0.001). During remission induction, patients on DFCI 95-01 had lower HRQL than patients on DFCI 00-01. Patients on DFCI 00-01 had ~0.2 and 0.3 fewer QALYs than controls, measured by HUI2 and HUI3, respectively. QALYs for DFCI 00-01 patients during treatment were similar to those for DFCI 95-01 patients. The trajectory of improvement in HRQL during the treatment of acute lymphoblastic leukemia in children was confirmed.

*Centre for Health Economics and Policy Analysis

Departments of Health Research Methods, Evidence, and Impact



¶¶Pediatrics, McMaster University

##McMaster Children’s Hospital, Hamilton

Health Utilities Inc., Dundas

∥∥Hospital for Sick Children, Toronto, ON

#Hôpital Sainte Justine, Montreal

††Le Centre Hospitalier de l’Universite Laval, Quebec City, QC, Canada

Dana-Farber Cancer Institute, and Boston Children’s Hospital, Boston, MA

**San Jorge Children’s Hospital, San Juan, Puerto Rico

‡‡Columbia Presbyterian Medical Center, New York, NY

§§Maine Children’s Cancer Program, Scarborough, ME

Supported by Canadian Cancer Society Research Institute (CCSRI) grant # 20400.

The authors declare no conflict of interest.

Reprints: Ronald Barr, MBChB, MD, Health Sciences Centre, Room 3N27, McMaster University, 1280 Main Street West, Hamilton ON, L8S 4J9, Canada (e-mail:

Received October 27, 2017

Accepted December 22, 2017

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