ArticleBlood advances2022
Morbidities and mortality in patients with hereditary thrombotic thrombocytopenic purpura.
Article in Blood advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed, 32 citations in OpenAlex.
- Estimating Long-Term Organ Damage Risk in Patients with Congenital Thrombotic Thrombocytopenic Purpura Receiving Recombinant ADAMTS13 Prophylaxis: A Structured Expert Elicitation Study.Advances in therapy · 2026Article
- Long Term Follow Up of Hereditary Thrombotic Thrombocytopenic Purpura on Plasma Therapy for 9 Years: A Case Report.Clinical case reports · 2026Article
- ADAMTS-13 R1206K variant exhibits an open conformation and an enhanced proteolytic activity toward von Willebrand factor.Journal of thrombosis and haemostasis : JTH · 2026Article
- The impact of hereditary TTP on psychological health, cognition, and well-being: a patient-reported outcomes study.Blood vessels, thrombosis & hemostasis · 2026Article
- Hereditary Thrombotic Thrombocytopenic Purpura Associated With Recurrent Strokes and Prominent Nervous System Involvement in a Young Chinese Female.Clinical case reports · 2026Article
- Natural History and Treatment Outcomes of Congenital Thrombotic Thrombocytopenic Purpura: A Retrospective Longitudinal Cohort Study.Journal of health economics and outcomes research · 2026Article
- Modern management of congenital thrombotic thrombocytopenic purpura (cTTP).Hematology. American Society of Hematology. Education Program · 2025Article
- Optimizing the management of congenital thrombotic thrombocytopenic purpura.Research and practice in thrombosis and haemostasis · 2025Article
- Thrombotic Thrombocytopenic Purpura in Pediatric Patients.Biomedicines · 2025Review
- Hereditary thrombotic thrombocytopenic purpura mimicking immune thrombocytopenia was revealed by miscarriage-novel compound heterozygous mutations in hTTP.BMC medical genomics · 2024Article
- Novel mechanisms of action of emerging therapies of hereditary thrombotic thrombocytopenic purpura.Expert review of hematology · 2024Review
- Examining an Interaction Between Plasma Exchange and Apixaban Resulting in an Acute Ischemic Stroke.The Journal of pharmacy technology : jPT : official publication of the Association of Pharmacy Technicians · 2024Article
- A Systematic Review of the Epidemiology and Disease Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.Journal of blood medicine · 2024Review
- Hereditary Thrombotic Thrombocytopenic Purpura.Genes · 2023Review
- Clinical Manifestations, Current and Future Therapy, and Long-Term Outcomes in Congenital Thrombotic Thrombocytopenic Purpura.Journal of clinical medicine · 2023Review
- [Clinical diagnosis and treatment of hereditary thrombocytopenia and purpura: a report of five cases and literature review].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2023Review
- Hereditary thrombotic thrombocytopenic purpura: The risk for death at birth.Research and practice in thrombosis and haemostasis · 2022Article
- Early indicators of neonatal-onset hereditary thrombotic thrombocytopenia purpura.Research and practice in thrombosis and haemostasis · 2022Article
- Article
- Aspirin prophylaxis for hereditary and acquired thrombotic thrombocytopenic purpura?American journal of hematology · 2022Article
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hereditary thrombotic thrombocytopenic purpura (hTTP) is a rare disorder caused by severe ADAMTS13 deficiency. Major morbidities and death at a young age are common. Although replacement of ADAMTS13 can prevent morbidities and death, current regimens of plasma prophylaxis are insufficient. We identified 226 patients with hTTP in 96 reports published from 2001 through 2020. Age at diagnosis was reported for 202 patients; 117 were female and 85 were male. The difference was caused by diagnosis of 34 women during pregnancy, suggesting that many men and nulliparous women are not diagnosed. Eighty-three patients had severe jaundice at birth; hTTP was suspected and effectively treated in only 3 infants. Of the 217 patients who survived infancy, 73 (34%) had major morbidities defined as stroke, kidney injury, or cardiac injury that occurred at a median age of 21 years. Sixty-two patients had stroke; 13 strokes occurred in children age 10 years or younger. Of the 54 patients who survived their initial major morbidity and were subsequently observed, 37 (69%) had sustained or subsequent major morbidities. Of the 39 patients who were observed after age 40 years, 20 (51%) had experienced a major morbidity. Compared with an age- and sex-matched US population, probability of survival was lower at all ages beginning at birth. Prophylaxis was initiated in 45 patients with a major morbidity; in 11 (28%), a major morbidity recurred after prophylaxis had begun. Increased recognition of hTTP and more effective prophylaxis started at a younger age are required to improve health outcomes.
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