Grants Awarded

Burden of morbidity and premature mortality in patients with Langerhans cell histiocytosis 

Principal Investigator:
Dr. Gaurav Goyal
University of Alabama
Birmingham, AL

Date of Award:
December 2022

Amount of Award:

Layperson Summary:
Langerhans cell histiocytosis (LCH) is a rare type of blood cancer that can affect any organ system of the body in children and adults. Thanks to improvements in treatments, some studies have suggested that the LCH-specific survival for patients with LCH may be improving in the recent years.  However, there is a lack of comprehensive studies defining the chronic medical conditions and long-term mortality experienced by patients with LCH.  Studies from our group have shown that there is a high incidence of acute leukemia and other cancers among LCH survivors.

In a recent analysis of a large, we found preliminary evidence for high risk of death among LCH patients from non-LCH-related causes (heart disease, lung disease, infections, second cancers, etc.). Chronic medical conditions may arise because of cancer treatments or due to the underlying LCH itself. Moreover, the chronic medical conditions can also lead to an adverse impact on the quality of life of people with LCH.  These studies show that there is a need to conduct a more definitive study to define the burden of medical conditions, quality of life, and cause-specific mortality in people with LCH. Additionally, there is also a need to define the prevalence of chronic pain and need for healthcare visits (clinic, hospital, emergency department) among people with LCH to gauge the healthcare needs of survivors.

We propose a study to address these questions utilizing a large registry of patients with LCH housed within the Histiocytosis Association, the largest non-profit organization committed to improving outcomes of patients and families affected by histiocytosis. We will administer a survey questionnaire capturing details of chronic health conditions, health related quality of life, cognitive/psychological function, pain intensity, and healthcare utilization and compare the outcomes to people without LCH or other cancers. We anticipate that the results from our study will be instrumental in counseling LCH patients, designing survivorship programs to reduce the burden of chronic medical conditions/pain and to conduct future research to improve patient outcomes.

Eventually, we plan to apply our research methodology to other histiocytic disorders like
Erdheim Chester disease and Rosai-Dorfman disease to define the health needs of this unique patient population.