Impact of Risk-Stratified Therapy on Health Status in Survivors of Childhood Acute Lymphoblastic Leukemia: A Report from the Childhood Cancer Survivor Study Open Access

Dixon, Stephanie (Summer 2019)

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Purpose To evaluate the impact of changes in risk-stratified therapy on health status among adult survivors of childhood acute lymphoblastic leukemia (ALL).

Methods We estimated the self-reported prevalence of adverse health status, including poor general or mental health, functional impairment, activity limitations, and cancer-related pain or anxiety among 5119 survivors of childhood ALL diagnosed from 1970-1999. Therapy combinations defined treatment groups representative of 1970s therapy (70s), standard- and high-risk 1980s and 1990s therapy (80sSR, 80sHR, 90sSR, 90sHR), and relapse/bone marrow transplant (R/BMT). To compare outcomes between groups, log-binomial models adjusted for clinical and demographic factors estimated prevalence ratios (PR) with 95% confidence intervals (CI).

Results Overall, survivors were more likely than siblings to report increased risk for all health status outcomes including poor general health (13.5% vs. 7.4%; PR 1.97 95% CI 1.73-2.24). Compared to 70s, 90sSR were less likely to report functional impairment (PR [95% CI] 0.55 [0.41-0.75]), activity limitations (0.63 [0.45-0.87]) and cancer-related pain (0.65 [0.45-0.94]); however, although a lower proportion reported poor general health (90sSR 12.2% vs 70s 16.5%), a 22% reduction in likelihood (PR 0.78 [0.59-1.03]), this was not statistically significant. Compared to 70s, 90sHR were less likely to report poor general health (0.61 [0.40-0.92]), functional impairment (0.61 [0.40-0.91]) and activity limitations (0.63 [0.40-0.98]). Compared to 70s, a higher proportion of survivors from R/BMT reported adverse health status outcomes, albeit PR was not statistically significantly different. Compared to siblings, 90sSR reported no difference in activity limitations (PR 1.25 [0.96-1.62]).

Conclusion Risk-stratified ALL therapy has succeeded in reducing risk for certain components of adverse health status compared to 70s. Further, 90sSR had no increase in risk for activity limitations compared to siblings.

Table of Contents

Chapter I: Introduction

Introduction                                                                                     1

Purpose of the study                                                                       8

Public health impact of the study                                                8

Goals of the study                                                            9

Chapter II: Manuscript

Abstract                                                                                            12

Introduction                                                                                     14

Methods                                                                                             15

Results                                                                                                19

Discussion                                                                                          23

Tables and Figures                                                                            28

Chapter III: Expanded Discussion and Public Health Implications

Expanded Discussion                                                                        38

Public Health Implications                                                              42

Future Directions                                                                               42

References                                                                                            43

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