ArticleThe patient2019
Patient Experience with Congenital (Hereditary) Thrombotic Thrombocytopenic Purpura: A Conceptual Framework of Symptoms and Impacts.
Article in The patient, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
What it found
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Who cites it
10 citing papers in PubMed, 16 citations in OpenAlex.
- Determining what matters most to patients: qualitative interview techniques and methodological considerations for concept saliency analysis and reporting.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- Current standards and novel concepts in thrombotic thrombocytopenic purpura.Frontiers in medicine · 2026Review
- Natural History and Treatment Outcomes of Congenital Thrombotic Thrombocytopenic Purpura: A Retrospective Longitudinal Cohort Study.Journal of health economics and outcomes research · 2026Article
- Optimizing the management of congenital thrombotic thrombocytopenic purpura.Research and practice in thrombosis and haemostasis · 2025Article
- Understanding data visualization techniques in qualitative studies used to develop and validate patient-reported outcome measures: a targeted literature review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Review
- Development of a new patient-reported outcome measure for complex cryptoglandular fistulas (20-Item complex cryptoglandular fistula questionnaireJournal of patient-reported outcomes · 2024Article
- ADAMTS13 in the New Era of TTP.International journal of molecular sciences · 2024Review
- A Systematic Review of the Epidemiology and Disease Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.Journal of blood medicine · 2024Review
- Psychometric evaluation of a patient-reported outcomes instrument for congenital thrombotic thrombocytopenic purpura.Journal of patient-reported outcomes · 2023Review
- TTP: From empiricism for an enigmatic disease to targeted molecular therapies.British journal of haematology · 2022Review
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectiveThrombotic thrombocytopenic purpura is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia and thrombocytopenia, with variable clinical manifestations (e.g., central nervous system, renal, gastrointestinal, and cardiac effects). This study's objective was to gain an in-depth understanding of patients' experiences with the congenital form of thrombotic thrombocytopenic purpura, including the most salient symptoms and impacts associated with congenital thrombotic thrombocytopenic purpura and its treatment.
methodsAn initial conceptual model of thrombotic thrombocytopenic purpura symptoms and impacts was derived from a targeted literature review, refined by interviews with expert hematologists, and further refined by concept elicitation telephone interviews with adults with congenital thrombotic thrombocytopenic purpura in the USA. Patients reported the duration, frequency, and severity experienced for each concept, and rated level of disturbance on a minimum to maximum scale of 0-10.
resultsInterviews were conducted with 11 patients (mean age, 38.2 years; range 21-52 years) in three waves (n = 4, n = 4, n = 3). The most salient symptoms (reported most frequently and rated by patients as most disturbing) were fatigue, headache, bruising, joint pain, muscular pain, forgetfulness, and difficulty communicating. The most salient impacts included diminished ability to work/study, financial distress, feeling depressed, feeling anxious, and mood swings. Patients' comments reflected the pervasive nature of congenital thrombotic thrombocytopenic purpura symptoms and impacts, and unmet treatment needs.
conclusionsThe final conceptual model, which includes salient symptoms and impacts of congenital thrombotic thrombocytopenic purpura and reflects the disease burden, was derived by integrating inputs from the literature review, expert opinion, and patient interviews, and will be used to develop a congenital thrombotic thrombocytopenic purpura-specific, patient-reported outcome instrument.
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