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Review
. 2024 Aug 14:15:363-386.
doi: 10.2147/JBM.S464365. eCollection 2024.

A Systematic Review of the Epidemiology and Disease Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura

Ping Du  1 , Tiffany Cristarella  2 , Camille Goyer  2 , Yola Moride  2   3
Affiliations
Review

A Systematic Review of the Epidemiology and Disease Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura

Ping Du et al. J Blood Med. .

Abstract

Congenital (cTTP) and immune-mediated (iTTP) thrombotic thrombocytopenic purpura are serious and rare clotting disorders resulting from a deficiency in the ADAMTS13 enzyme. A systematic review was conducted using the Ovid® MEDLINE & Embase databases to synthesize the epidemiology and burden of cTTP and iTTP worldwide (from January 1, 2010, to February 6, 2020, with an update that covered the period January 1, 2020-February 11, 2022). Outcomes of interest were incidence and prevalence of TTP, incidence of acute episodes, mortality, burden of illness (eg complications, healthcare utilization, patient-reported outcomes) and disease management. A total of 221 eligible observational studies were included. The incidence rate of acute episodes ranged from 0.19-0.35 person-years in adult patients with cTTP, and 1.81-3.93 per million persons per year for iTTP in the general population. Triggers of acute episodes were similar for cTTP and iTTP, with pregnancy and infection the most commonly observed. Exacerbation in patients with iTTP varied widely, ranging from 2.4-63.1%. All-cause mortality was observed in 0-13.4% of patients with cTTP, across studies and follow-up periods, and in 1.1% (median follow-up: 0.4 years) to 18.8% (1 year) of patients with iTTP during acute episodes. Cardiovascular, renal, and neurological disease were common complications. TTP also led to work disturbances, feelings of anxiety and depression, and general activity impairment. TTP treatment regimens used were generally reflective of current treatment guidelines. The evidence identified describes a high patient burden, highlighting the need for effective treatment regimens leading to improvements in outcomes. Considerable evidence gaps exist, particularly for disease epidemiology, patient-reported outcomes, costs of disease management, and associated healthcare resource utilization. This review may help increase disease awareness and highlights the need for additional real-world studies, particularly in geographical regions outside the United States and Western Europe.

Keywords: ADAMTS13; burden of illness; disease management; epidemiology; patient-reported outcomes; thrombotic thrombocytopenic purpura.

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Conflict of interest statement

Ping Du was an employee of Takeda Development Center Americas, Inc. at the time of review development and a Takeda stockholder. Tiffany Cristarella and Camille Goyer are employees of YolaRX Consultants and received fees from Shire Human Genetic Therapies, Inc., a Takeda company, for conducting this analysis. Yola Moride is president of YolaRX Consultants and received fees from Shire Human Genetic Therapies, Inc., a Takeda company, for conducting this analysis. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
PRISMA flow chart for the selection of sources.
Figure 2
Figure 2
The incidence (A) and prevalence (B) of unspecified TTP, cTTP and iTTP.
See this image and copyright information in PMC

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