Date of Award

January 2018

Document Type

Thesis

Degree Name

Medical Doctor (MD)

Department

Medicine

First Advisor

Nina S. Kadan-Lottick

Abstract

With research and clinical advances over the past several decades, survival rates for childhood cancer have improved to greater than 80%. However, the path to survivorship poses a substantial burden to patients and families, with lengthy treatment courses requiring frequent hospital visits, and with late complications of treatment that can present years after therapy ends. This thesis explores each of these areas: one study investigating bone health and morbidity in leukemia and lymphoma survivors, and another study examining the cost burden to families of children during treatment for average-risk acute lymphoblastic leukemia (ALL). We aimed to determine the utility of surveillance, describe the burden to patients and families, and identify groups at highest risk who may benefit from intervention.

We conducted a retrospective cross-sectional study of bone mineral density (BMD) surveillance in childhood cancer survivors attending survivorship clinics at Yale-New Haven Hospital and Seattle Children’s Hospital. This study found a substantial yield of survivors with BMD more than one standard deviation below norms, with higher yield of low lumbar spine BMD in survivors of white race, with low body mass index, or who were older at diagnosis, and higher yield of low total body BMD in survivors exposed to cranial radiation, with growth hormone deficiency after treatment, or with low body mass index. Survivors who had fractures post-therapy had a significantly lower BMD, but the absolute difference was not clinically meaningful. Our findings suggest that lumbar spine BMD surveillance has the greatest utility in survivors diagnosed at 10 years of age or older, and total body BMD surveillance may be best utilized in patients diagnosed at less than 10 years of age, as well as survivors exposed to cranial radiation or with growth hormone deficiency

We conducted a prospective longitudinal study of financial toxicity in families of children undergoing treatment for average-risk ALL, which described a substantial educational, occupational, and financial cost burden that is highest early in treatment, but that persists until end of therapy. Occupational and opportunity costs are most noteworthy, with a sizable proportion of caregivers quitting or losing jobs, or not taking employment opportunities during their child’s ALL treatment.

As the population of childhood cancer survivors continues to grow, it becomes increasingly important to understand the burden posed to survivors and families during and after therapy and identify groups at the greatest risk of higher costs and late complications. Doing so may improve patient outcomes and quality of life.

Comments

This thesis is restricted to Yale network users only. It will be made publicly available on 05/29/2021

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