Abstract: SA-PO920
Ravulizumab Is Associated with Positive Clinical and Quality-of-Life Outcomes in Patients with Atypical Hemolytic Uremic Syndrome in a Real-World Setting
Session Information
- Glomerular Diseases: Therapeutics
November 04, 2023 | Location: Exhibit Hall, Pennsylvania Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Trials
Authors
- Wang, Yan, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Gatta, Francesca, Alexion Pharma GmbH, AstraZeneca Rare Disease, Zurich, Switzerland
- Ong, Moh-Lim, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
- Gibson, Gregor, Adelphi Real World, Bollington, United Kingdom
- Wynne-Cattanach, Kieran S., Adelphi Real World, Bollington, United Kingdom
- Conyers, Joe, Adelphi Real World, Bollington, United Kingdom
- Myren, Karl-Johan, Alexion, AstraZeneca Rare Disease, Stockholm, Sweden
Background
Ravulizumab (RAV) is a complement C5 inhibitor first approved in 2019 for the treatment of atypical hemolytic uremic syndrome (aHUS), a rare disease characterized by hemolytic anemia, thrombocytopenia and organ damage. This study assessed real-world clinical and quality of life (QoL) outcomes in patients with aHUS treated with RAV.
Methods
Data were drawn from the Adelphi aHUS Disease Specific ProgrammeTM, a cross-sectional survey of physicians reporting on their consulting patients with aHUS (data collected August–December 2022). This analysis included physician-reported, patient data from Germany, Italy, Spain, the UK, and the USA. Patients included had physician-diagnosed aHUS and were treated with RAV at time of survey.
Results
Overall, 65 patients were included: 31 had switched from eculizumab (ECU; switch cohort) and 34 had not received ECU before RAV initiation (naive cohort). Median (range) age at time of survey was 34 (15–75) and 39 (14–66) years in the switch and naive cohorts, respectively; 10% (n = 29) and 16% (n = 32) had family history of kidney disease. For switch and naive cohorts, respectively, median (interquartile range) time from aHUS diagnosis was 531 (233–1043) and 257 (152–456) days and RAV treatment duration was 163 (93–293) and 201 (106–476) days; prior ECU treatment duration was 150 (62–440) days (switch cohort only; n = 24). Two patients (6%) in the switch cohort had had a kidney transplantation before switching. Clinical outcomes are shown in the Table. Physician-reported overall QoL was rated ‘good’ to ‘excellent’ in 97% (overall physical/mental health: 97%/93%) and 82% (overall physical/mental health: 82%/85%) of patients in the switch and naive cohorts, respectively.
Conclusion
After initiation of RAV, the majority of patients had better clinical outcomes (compared with before RAV initiation) and a favorable QoL.
| Clinical outcomes | Switch cohort (n = 31) | Naive cohort (n = 34) |
| Patients undergoing dialysis, n (%) Before RAV Most recent/at time of survey | 5 (16) 1 (3) | 8 (24) 1 (3) |
| Platelet counts, n Patients with counts within normal range (150x109–400x109 cells/L), n (%) Before RAV Most recent/at time of survey | 23 10 (44) 16 (70) | 27 3 (11) 11 (41) |
| Lactate dehydrogenase levels, n Patients with levels below 1.5x upper limit of normal range, n (%) Before RAV Most recent/at time of survey | 11 6 (55) 11 (100) | 17 4 (24) 11 (65) |
| Serum creatinine levels, n Serum creatinine, mean, mg/dL Before RAV Most recent/at time of survey | 19 2.1 1.5 | 17 3.4 1.7 |
Funding
- Commercial Support – Alexion, AstraZeneca Rare Disease. The DSP is an Adelphi Real World product. Alexion were a subscriber to the DSP and did not influence the original survey through either contribution to the design of questionnaires or data collection.