Hereditary thrombotic thrombocytopenic purpura and COVID‐19: Impacts of vaccination and infection inrare disease

Tarasco, Erika; Krogh, Anne Sophie von; Hrdličková, Radomíra; Braschler, Thomas; Iwaniec, Teresa; Knöbl, Paul; Hamada, Eriko; Pikovsky, Oleg; Farese, Stefan; Gutwein, Odit; Kessler, Petr; Schultz, Nina Haagenrud; Auer, Charis von; Windyga, Jerzy; Friedman, Kenneth D.; Hrachovinová, Ingrid; George, James N.; Matsumoto, Masanori; Schneppenheim, Reinhard; Lämmle, Bernhard; Hovinga, Johanna A. Kremer

doi:10.1002/rth2.12814

Cited by 3 publications

(1 citation statement)

References 24 publications

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“…So far, information on the long-term clinical course, organ involvement, and complications of cTTP is limited. Gradually, data are beginning to appear from the International Hereditary TTP Registry (www.ttpregistry.net, ClinicalTrials.gov identifier NCT01257269; a multinational cohort study with global recruitment 67,74,78,79 ) and larger national cTTP cohorts from France, 63,70,72 the United Kingdom, 71,80 Israel, 65,77 Japan, 62,76,81,82 and Norway. 64,83 The latter three countries have enrolled (part of) their patients into the International Hereditary TTP Registry.…”

Section: Ttp Diagnosis Based On Adamts13 Assaysmentioning

confidence: 99%

100 Years of Thrombotic Thrombocytopenic Purpura: A Story of Death and Life

Lämmle,

Vanhoorelbeke,

Kremer Hovinga

et al. 2024

Hamostaseologie

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One hundred years ago, in 1924, the first description of a patient with a disease, now known as thrombotic thrombocytopenic purpura (TTP) was published by Dr. Eli Moschcowitz. In honor of this report, this article, written by distinguished specialists in TTP, reviews the increase in scientific knowledge on this disease during the last 100 years. It covers the scientific progress from plasma therapy, the first beneficial treatment for TTP, to the elucidation of the pathophysiology, the discovery of ADAMTS13, the development of assays and targeted therapies up to the modern treatment concepts, that improved the outcome of TTP from an incurable disease to a well understood and treatable disorder.

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Section: Ttp Diagnosis Based On Adamts13 Assaysmentioning

confidence: 99%

100 Years of Thrombotic Thrombocytopenic Purpura: A Story of Death and Life

Lämmle,

Vanhoorelbeke,

Kremer Hovinga

et al. 2024

Hamostaseologie

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Elasomeran/plasma/tozinameran

2023

Reactions Weekly

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Impact of the COVID-19 Pandemic on People Living With Rare Diseases and Their Families: Results of a National Survey

Macaluso,

Rothenberg,

Ferkol

et al. 2024

JMIR Public Health Surveill

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Background With more than 103 million cases and 1.1 million deaths, the COVID-19 pandemic has had devastating consequences for the health system and the well-being of the entire US population. The Rare Diseases Clinical Research Network funded by the National Institutes of Health was strategically positioned to study the impact of the pandemic on the large, vulnerable population of people living with rare diseases (RDs). Objective This study was designed to describe the characteristics of COVID-19 in the RD population, determine whether patient subgroups experienced increased occurrence or severity of infection and whether the pandemic changed RD symptoms and treatment, and understand the broader impact on respondents and their families. Methods US residents who had an RD and were <90 years old completed a web-based survey investigating self-reported COVID-19 infection, pandemic-related changes in RD symptoms and medications, access to care, and psychological impact on self and family. We estimated the incidence of self-reported COVID-19 and compared it with that in the US population; evaluated the frequency of COVID-19 symptoms according to self-reported infection; assessed infection duration, complications and need for hospitalization; assessed the influence of the COVID-19 pandemic on RD symptoms and treatment, and whether the pandemic influenced access to care, special food and nutrition, or demand for professional psychological assistance. Results Between May 2, 2020, and December 15, 2020, in total, 3413 individuals completed the survey. Most were female (2212/3413, 64.81%), White (3038/3413, 89.01%), and aged ≥25 years (2646/3413, 77.53%). Overall, 80.6% (2751/3413) did not acquire COVID-19, 2.08% (71/3413) acquired it, and 16.58% (566/3413) did not know. Self-reported cases represented an annual incidence rate of 2.2% (95% CI 1.7%-2.8%). COVID-19 cases were more than twice the expected (71 vs 30.3; P<.001). COVID-19 was associated with specific symptoms (loss of taste: odds ratio [OR] 38.9, 95% CI 22.4-67.6, loss of smell: OR 30.6, 95% CI 17.7-53.1) and multiple symptoms (>9 symptoms vs none: OR 82.5, 95% CI 29-234 and 5-9: OR 44.8, 95% CI 18.7-107). Median symptom duration was 16 (IQR 9-30) days. Hospitalization (7/71, 10%) and ventilator support (4/71, 6%) were uncommon. Respondents who acquired COVID-19 reported increased occurrence and severity of RD symptoms and use or dosage of select medications; those who did not acquire COVID-19 reported decreased occurrence and severity of RD symptoms and use of medications; those who did not know had an intermediate pattern. The pandemic made it difficult to access care, receive treatment, get hospitalized, and caused mood changes for respondents and their families. Conclusions Self-reported COVID-19 was more frequent than expected and was associated with increased prevalence and severity of RD symptoms and greater use of medications. The pandemic negatively affected access to care and caused mood changes in the respondents and family members. Continued surveillance is necessary.

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Hereditary thrombotic thrombocytopenic purpura and COVID‐19: Impacts of vaccination and infection inrare disease

Cited by 3 publications

References 24 publications

100 Years of Thrombotic Thrombocytopenic Purpura: A Story of Death and Life

100 Years of Thrombotic Thrombocytopenic Purpura: A Story of Death and Life

Elasomeran/plasma/tozinameran

Impact of the COVID-19 Pandemic on People Living With Rare Diseases and Their Families: Results of a National Survey

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