Uncovering Root Causes Underlying Gaps in the Evaluation and Integration of the Latest Clinical Evidence in Congenital Thrombotic Thrombocytopenic Purpura: Key Findings from a National Hematology Care Team Survey
Authors: Borogovac Azra , Moses-Okwei Ellie , Gurska Lindsay , Zyborowicz Emily , Sullivan Shelby , Carter Jeffrey , Heggen Cherilyn 
Published: December 2024

Background
Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening disorder marked by a deficiency in ADAMTS13 activity, affecting about one in a million people. Without prompt treatment, cTTP can cause significant morbidity and mortality. With the emergence of novel treatment options, enhancing physician awareness and recognition of cTTP, as well as appropriate integration of the latest clinical data into diagnosis and management is essential to improve outcomes. To identify specific barriers to the uptake and integration of new clinical evidence in cTTP, PRIME conducted a national survey of hematology providers assessing their awareness of proper diagnosis, management, and emerging clinical evidence in cTTP.
Methods
Baseline surveys were disseminated to 135 hematology healthcare providers (HCPs), with questions designed to assess challenges and barriers faced in diagnosis, management, and integration of the latest evidence in cTTP. Among the respondents, 68% were hematologists, 8% nurse practitioners (NP) or physician's assistants (PA), 12% nurses, and 6% pharmacists, with an average of 18 years caring for patients with hematologic disorders. Respondents reported encountering approximately 2,978 cTTP patients in their practice, averaging 28 patients per provider.
Results
Gaps were observed in providers' confidence in their ability to recognize symptoms of cTTP (63% reported high/very high confidence), obtain a differential diagnosis of cTTP (69%), and optimally treat and manage patients with cTTP (66%). Reported barriers to prompt diagnosis include differentiating cTTP from other potential diagnoses based on patient presentation (70%), lack of ADAMTS13 activity testing availability (53%), lack of a referral network (43%), and unfamiliarity with cTTP symptoms and disease characteristics (42%). Furthermore, top challenges in managing cTTP involve engaging patients in discussions and incorporating their goals and preferences into treatment plans (56%), managing acute complications of TTP episodes (51%), deficiencies in knowledge of cTTP and treatment and management options (47%), and preventing TTP relapses (46%).
Regarding limitations to current treatment options, 61% cited time requirements to complete treatment infusions, and 58% cited limited availability of centers performing treatment infusions. HCP knowledge of emerging evidence regarding new rADAMTS13 therapy was low, with only 26% identifying accurate information from the pivotal phase 3 281102 clinical trial. Anticipated challenges in integrating new cTTP therapies include medications not being on formulary or covered by patients' insurance (68%) and high medication costs preventing patients from affording copays (51%). Providers reported the most helpful strategies for integrating new cTTP therapies involve education on new and emerging clinical evidence for cTTP treatment and management (61%), improving efficiency of obtaining insurance approval/prior authorization for novel therapies (59%), development of clinic- or system-level processes and protocols to support evidence-based testing and treatment integration (45%), education on identifying patients eligible for prophylactic therapy (43%), and improving patient engagement in discussion of new treatment options and shared decision-making (43%).
Conclusions
HCPs disclosed key educational barriers that limit their ability to accurately diagnose, manage acute complications, and engage patients with cTTP. Notably, difficulties in differentiating cTTP from other diagnoses, limited availability of testing, and managing the costs and insurance coverage of new therapies were highlighted. To optimize cTTP care, educational programming focused on integrating new clinical evidence for diagnosis and management of cTTP, including adoption of new therapies, and improving patient engagement strategies is critical. These insights can guide future initiatives to bridge persistent gaps in quality cTTP care.
Study Sponsor Statement
The study reported in this abstract was supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc., who had no role in the study design, execution, analysis, or reporting.