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Follow-up Evaluation of Patients with Hereditary Thrombotic Thrombocytopenic Purpura (hTTP) from the International Hereditary TTP Registry: Incidence of Acute Disease Episodes with or without Regular Prophylactic Plasma Infusions

E. Tarasco1,2, L. Bütikofer3, G. Sinkovits4, Z. Prohászka4, A.S. von Krogh5, P.N. Knöbl6, K.D. Friedman7, J.N. George8, I. Aebi-Huber1,2, K.A. Jalowiec1, Z. Cermakova9, M. Górska-Kosicka10, J. Windyga10, I. Hrachovinova11, C.R. Largiadèr12, M. Matsumoto13, B. Lämmle1,14, J.A. Kremer Hovinga1,2

1Inselspital, University Hospital of the University of Bern, Department of Hematology and Central Hematology Laboratory, Bern, Switzerland, 2University of Bern, Department for BioMedical Research (DBMR), Bern, Switzerland, 3University of Bern, CTU, Bern, Switzerland, 4Research Laboratory, 3rd Department of Internal Medicine and MTA-SE Research Group of Immunology and Hematology, Budapest, Hungary, 5St Olavs Hospital Trondheim University Hospital, Department of Hematology, Trondheim, Norway, 6University of Vienna, Division of Hematology and Hemostasis, Department of Medicine 1, Vienna, Austria, 7Medical College of Wisconsin, Division of Hematology and Oncology, Milwaukee, United States, 8University of Oklahoma Health Sciences Center, Department of Biostatistics and Epidemiology, College of Public Health, Oklahoma City, United States, 9University Hospital Ostrava, Blood Center, Ostrava, Czech Republic, 10Institute of Hematology and Transfusion Medicine, Department of Disorders of Hemostasis and Internal Medicine, Warsaw, Poland, 11Institute of Hematology and Blood Transfusion, NRL for Hemostasis, Prague, Czech Republic, 12Inselspital, University Hospital of the University of Bern, Bern, Switzerland, 13Nara Medical University, Department of Blood Transfusion Medicine, Nara, Japan, 14University Medical Center Mainz, Center for Thrombosis and Hemostasis, Mainz, Germany

Abstract Number: PB1872

Meeting: ISTH 2020 Congress

Theme: Thrombotic Microangiopathies » ADAMTS13 and TTP

Background: Hereditary Thrombotic Thrombocytopenic Purpura (hTTP) is an ultra-rare autosomal-recessively inherited disorder characterized by recurring acute episodes of thrombotic microangiopathy. Patients have a variable disease course and little is known about the annual rate of acute episodes.

Aims: This study reports the incidence of acute episodes in hTTP patients enrolled in the International Hereditary TTP Registry during follow-up.

Methods: At the end of 2019, we had enrolled 139 confirmed hTTP patients. Prospective follow-up data of 87 hTTP patients from enrollment until 12/2019 were analyzed.

Results: The 87 hTTP patients were followed for 4.2 years (median; range 0-15 years). The median age at overt disease onset and at clinical diagnosis was 4.6 years (range 0-70) and 18 years (range 0-70), respectively.18 female and 25 male patients received regular prophylactic plasma infusions, 11 females and 14 males did not. Prophylaxis was temporarily withheld in 12 females and 6 males (Figure).
109 acute episodes were reported during follow-up in 41/87 patients: 74 in patients with and 35 in patients without prophylaxis. This results in annual episode rates/patient-year of 0.44 (95%CI 0.33-0.58) and 0.16 (95%CI 0.10-0.24) in female and male patients under prophylaxis, respectively; and of 0.38 (95%CI 0.24-0.57) and 0.23 (95%Cl 0.12-0.41) in females and males not under prophylaxis, respectively.
We prospectively followed 13 pregnancies in 5 patients: 6 in 3 women under plasma prophylaxis installed before pregnancy, and 7 in 2 women receiving plasma infusion only during pregnancy. Five acute episodes occurred resulting in one abortion.

Conclusions: Current prophylactic plasma infusions (about 1-3 plasma units every 1-4 weeks) are often insufficient to prevent acute episodes. Patients under prophylaxis have a similar incidence of acute episodes as those not under prophylaxis. Females seem to suffer more frequent acute episodes than males, not attributable to pregnancy alone. Further prospective follow-up will provide robustness of our estimates.

To cite this abstract in AMA style:

Tarasco E, Bütikofer L, Sinkovits G, Prohászka Z, von Krogh AS, Knöbl PN, Friedman KD, George JN, Aebi-Huber I, Jalowiec KA, Cermakova Z, Górska-Kosicka M, Windyga J, Hrachovinova I, Largiadèr CR, Matsumoto M, Lämmle B, Kremer Hovinga JA. Follow-up Evaluation of Patients with Hereditary Thrombotic Thrombocytopenic Purpura (hTTP) from the International Hereditary TTP Registry: Incidence of Acute Disease Episodes with or without Regular Prophylactic Plasma Infusions [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/follow-up-evaluation-of-patients-with-hereditary-thrombotic-thrombocytopenic-purpura-http-from-the-international-hereditary-ttp-registry-incidence-of-acute-disease-episodes-with-or-without-regular/. Accessed August 15, 2022.

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