PB0700 - Identifying Unmet Needs Related to Treatment and Disease Control in Immune Thrombocytopenia (ITP): US Patient Survey

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Nichola Cooper

Consultant Hematologist
Hammersmith Hospital
London, England, United Kingdom

Co-Author(s)

CK

Caroline Kruse

President and CEO
Platelet Disorder Support Association
Cleveland, Ohio, United States
SD

Sharon Deneen Morgan

Volunteer and ITP Patient
PDSA
Memphis, Tennessee, United States
MA

Marleni Arvelo-Saillant

Global Public Affairs
Sanofi
Paris, Ile-de-France, France
JR

Jean-Pascal Roussy

Global Public Affairs
Sanofi
New York, New Jersey, United States
MC

Matias Cordoba

Global Medical Director
Sanofi
Cambridge, Massachusetts, United States
IG

Imene Gouia

Health Economics and Value Assessment
Sanofi
Gentilly, Ile-de-France, France
LS

Lisa-Anne Schmitt

US Public Affairs
Sanofi
Cambridge, Massachusetts, United States
ER

Erin Reineke

Scientific Communications
Sanofi
Cambridge, Massachusetts, United States
TG

Terry Gernsheimer

Division of Hematology
University of Washington and Fred Hutchinson Cancer Center
Seattle, Washington, United States

Background: Chronic disease, increased comorbidities, elevated bleeding risks, and impaired HRQOL may negatively affect ITP patients.

Aims: Assess patient experience, unmet needs, and implications of ITP disease management and control.

Methods: A cross-sectional, IRB-approved survey was conducted by a Sanofi and Platelet Disorder Support Association collaboration in US patients (≥18 years) with ITP diagnosed for ≥1 year. All patients provided informed consent.

Results: From Feb2023–Apr2023, 80 ITP patients responded to an online questionnaire. Most were aged >60 years (54%), female (79%), and had primary (70%) vs secondary (30%) ITP; 49% reported ≥2 comorbidities (most common was autoimmune disease, 46%). Most common ITP symptoms were fatigue (≥68%) and bruising (62%).

For 61% of patients currently receiving ITP therapy, these included TPO-RA (39%), CS (15%), IVIg (8%), and/or rituximab (8%). For those not receiving treatment, median time since last ITP therapy was 3 years (IQR, 0.5-5.8). Overall, 50% of patients changed treatment ≥4 times during their care path mainly due to lack of efficacy. Despite changes, 75% declared good adherence and satisfaction to prescribed treatment. Highest ranked factors for controlling ITP included increasing/stable platelet counts and decreasing fatigue and bleeding severity (Figure 1). Independent of treatment satisfaction, 92% were involved with their disease management, 88% felt informed, and 83% trusted their physician.

Although 25% felt their ITP was well-managed, 46% desired less fatigue and anxiety related to platelet counts and bleeding, main indicators for poor ITP control and worsened HRQOL. Of 50 respondents indicating poor ITP control, most worrisome aspects were potentially severe symptoms and limited daily tasks/physical activities (Figure 2). Fears of worsening ITP (49%) and long-term side effects (33%) contributed to concerns about long-term disease control.

Conclusion(s): Patients with ITP are looking for long-term disease management treatment options that improve their platelet counts and reduce their fatigue and anxiety.