Patient-Reported Outcomes and Daily Activity Assessed with a Digital Wearable Device (FITBIT™) in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab: The Reveal Study
Author(s)
Griffiths EA1, Wang A2, Tomazos I3, Min JS4, Lee WN5, Patel Y2, Yu J2, Dingli D6
1Roswell Park Comprehensive Cancer Center, Buffalo, NY, USA, 2Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 3Alexion, AstraZeneca Rare Disease*, Boston, MA, USA, 4Evidation Health*, San Mateo, CA, USA, 5Evidation Health, San Mateo, CA, USA, 6Mayo Clinic, Rochester, MN, USA
Presentation Documents
OBJECTIVES: To characterize patient experiences during ravulizumab or eculizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH) using a novel real-world data set collected from a consumer wearable device and electronically collected PROs (ePROs).
METHODS:
This 32-week prospective, non-interventional, observational cohort study enrolled adult patients with PNH in the USA. ePRO data were collected weekly by survey and included Patient-Reported Outcomes Measurement Information System (PROMIS)-Physical Health, PROMIS-Mental Health and Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. Continuous activity data were collected via Fitbit™ and included physical activity, sleep pattern and heart rate. Results were compared with general US population normal values; exploratory analyses were performed to correlate activity data with ePROs.RESULTS:
Among the 28 patients with PNH receiving ravulizumab enrolled in the study, median (range) age was 28 (15–62) years at PNH diagnosis and 34 (19–64) years at baseline. Most recent self-reported mean (standard deviation [SD]) clone size (type unknown) was 63% (36%), hemoglobin level 11.22 (1.77) g/dL and lactate dehydrogenase level 246 (89) U/L. Over the 32-week study period, patients treated with ravulizumab had mean (SD) ePRO scores and activity data within the general US population normal range: PROMIS-Physical Health, 51 (7); PROMIS-Mental Health, 51 (8); FACIT-Fatigue, 40 (9); daily steps, 7476 (3164); sleep duration, 7.7 (1.3) hours; and resting heart rate, 67 (8) bpm. Higher daily steps were positively correlated with PROMIS-Physical Health (R=0.12; q<0.05) and PROMIS-Mental Health (R=0.12; q<0.05). Lower resting heart rate was positively correlated with PROMIS-Physical Health (R=0.12; q<0.05).CONCLUSIONS:
In this pilot study, patients with PNH who were clinically stable on ravulizumab had physical and mental health, fatigue and daily activity similar to the general US population. Higher daily steps and lower resting heart rate were positively correlated with increased quality of life among ravulizumab-treated patients with PNH.Conference/Value in Health Info
2023-05, ISPOR 2023, Boston, MA, USA
Value in Health, Volume 26, Issue 6, S2 (June 2023)
Code
SA70
Topic
Study Approaches
Topic Subcategory
Prospective Observational Studies
Disease
No Additional Disease & Conditions/Specialized Treatment Areas