A scale for patient-reported symptom assessment for patients with Erdheim-Chester disease Journal Article
| Authors: | Diamond, E. L.; Reiner, A. S.; Buthorn, J. J.; Shuk, E.; Applebaum, A. J.; Hyman, D. M.; Abdel-Wahab, O.; Rampal, R.; Janku, F.; Brewer, K.; Campbell, J.; Mao, J. J.; Atkinson, T. M.; Panageas, K. S. |
| Article Title: | A scale for patient-reported symptom assessment for patients with Erdheim-Chester disease |
| Abstract: | Erdheim-Chester disease (ECD) is an ultra-rare hematologic neoplasm characterized by somatic mutations of the MAPK pathway and by accumulation of lesional histiocytes within tissues. Clinical phenotypes and sites of disease involvement are heterogenous in ECD, and no tool exists for systematic and comprehensive assessment of ECD symptomatology. We describe a collaborative effort among ECD specialists, patient-reported outcome (PRO) methodologists, and ECD patients to develop the Erdheim-Chester Disease Symptom Scale (ECD-SS): a symptom inventory for clinical ECD care and evaluation of ECD therapies. Methodologically rigorous focus groups led to the identification of 63 ECD symptoms in 6 categories, incorporated into the ECD-SS with respect to both severity and frequency. Among 50 ECD patients participating in a prospective registry study completing the ECD-SS, 46 (92%) reported neurological/psychological symptoms, 29 (58%) reported pain, and at least one-half reported mood symptoms, memory problems, or fatigue. Symptoms were highly frequent or almost constant regardless of their severity. The ECD-SS is a rigorously developed, patient-centered tool that demonstrates the wide and previously unappreciated burden of symptomatology experienced by ECD patients. Further studies will refine the symptom inventory and define its psychometric properties and role in clinical care and investigation in the context of ECD. © 2019 by The American Society of Hematology. |
| Keywords: | adult; clinical article; aged; fatigue; pain; cohort analysis; patient care; disease severity; medical specialist; rating scale; personal experience; mood disorder; patient-reported outcome; disease burden; human; male; female; priority journal; article; erdheim chester disease |
| Journal Title: | Blood Advances |
| Volume: | 3 |
| Issue: | 7 |
| ISSN: | 2473-9529 |
| Publisher: | American Society of Hematology |
| Date Published: | 2019-04-09 |
| Start Page: | 934 |
| End Page: | 938 |
| Language: | English |
| DOI: | 10.1182/bloodadvances.2018030502 |
| PUBMED: | 30917949 |
| PROVIDER: | scopus |
| PMCID: | PMC6457219 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 2 August 2019 -- Source: Scopus |
Altmetric
Citation Impact
BMJ Impact Analytics
MSK Authors
Related MSK Work