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Abstract: SA-PO921

Treatment Satisfaction with Ravulizumab in Patients with Atypical Hemolytic Uremic Syndrome in a Real-World Setting

Session Information

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
  • Myren, Karl-Johan, Alexion, AstraZeneca Rare Disease, Stockholm, Sweden
  • 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
  • Ong, Moh-Lim, Alexion, AstraZeneca Rare Disease, Boston, Massachusetts, United States
Background

Atypical hemolytic uremic syndrome (aHUS) is a rare disease characterized by hemolytic anemia, thrombocytopenia, and organ damage. Ravulizumab (RAV) is a complement C5 inhibitor approved for the treatment of aHUS in 2019. Our study assessed real-world RAV treatment satisfaction.

Methods

Data were drawn from the Adelphi aHUS Disease Specific ProgrammeTM, a cross-sectional survey of physicians and their patients with aHUS (Aug–Dec 2022) in Europe and the USA. Physicians reported broad attitudinal data and demographics, treatment history, and treatment satisfaction for their patients with aHUS. Patients could report their treatment satisfaction in a separate survey.

Results

Overall, 53 physicians who completed the survey had experience with RAV (EU5 [58%], USA [42%]), most were nephrologists (53%) practicing in urban areas/major city centers (66%) with a median of 11 (range: 1–22) years of experience treating patients with aHUS. Of the surveyed physicians, 87% were ‘completely satisfied' or ‘satisfied’ with RAV; no physicians were ‘dissatisfied’ or ‘completely dissatisfied’. In total, 65 patients currently treated with RAV were included: 31 switched from eculizumab (ECU; switch cohort) and 34 did not receive ECU prior to RAV initiation (naive cohort). In the switch and naive cohorts, respectively, median RAV treatment duration was 163 (interquartile range: 93–293) and 201 (106–476) days; and time since aHUS diagnosis was 531 (233–1043) and 257 (152–456) days. Data from patient records showed high physician satisfaction with RAV (Table). In 84% of patients in the switch cohort, physicians reported higher satisfaction with RAV vs ECU (n=26; top reasons were fewer infusions required [92%] and less burdensome to patient/caregiver [54%]); no physicians reported higher satisfaction with ECU. From the patient survey, RAV satisfaction was 100% (switch cohort; n=8) and 83% (naive cohort; n=11).

Conclusion

There was concordance in high satisfaction with RAV between physicians and patients, regardless of prior ECU treatment. No dissatisfaction with RAV was reported.

 Switch cohort
(n=30)a
Naive cohort
(n=34)
Physician satisfaction with RAV from patient recordsb, n (%)
‘Completely satisfied’
‘Satisfied’
‘Neither satisfied or dissatisfied’
‘Dissatisfied’
‘Completely dissatisfied’
10 (33)
19 (63)
1 (3)
0
0
13 (38)
19 (56)
2 (6)
0
0
an=1 patient excluded as RAV initiation was on the same day as the survey; bPhysycians were asked to rate their satisfaction with RAV regarding the treatment of specific patients with aHUS based on patient records.

Funding

  • Commercial Support – Alexion, AstraZeneca Rare Disease. The Disease Specific ProgrammeTM is an Adelphi Real World product. Alexion was a subscriber to the Disease Specific ProgrammeTM and did not influence the original survey through either contribution to the design of questionnaires or data collection.