Purpura fulminans in severe congenital protein C deficiency: Monitoring of treatment with protein C concentrate

1996 ◽  
Vol 155 (1) ◽  
pp. 20-25 ◽  
Author(s):  
F. -M. Müller ◽  
W. Ehrenthal ◽  
G. Hafner ◽  
D. Schranz
Keyword(s):  
Protein C ◽  
Purpura Fulminans ◽  
Protein C Deficiency ◽  
Protein C Concentrate ◽  
Congenital Protein C Deficiency

Purpura Fulminans in a Chinese Boy With Congenital Protein C Deficiency

PEDIATRICS ◽  
1986 ◽  
Vol 77 (5) ◽  
pp. 670-676
Author(s):  
Patrick Yuen ◽  
Alfred Cheung ◽  
Hsiang Ju Lin ◽  
Faith Ho ◽  
Jun Mimuro ◽  
...  
Keyword(s):  
Disseminated Intravascular Coagulation ◽  
Protein C ◽  
Purpura Fulminans ◽  
Fresh Frozen Plasma ◽  
Protein C Deficiency ◽  
Intravascular Coagulation ◽  
The Family ◽  
Fresh Frozen ◽  
Frozen Plasma ◽  
Congenital Protein C Deficiency

Severe and recurrent purpura fulminans developed in a Chinese boy at one day of age. Results of coagulation studies performed on the patient during attacks were compatible with the diagnosis of disseminated intravascular coagulation. Subsequent investigations have revealed that the patient is homozygous and that his parents are heterozygous for protein C deficiency. Cryoprecipitate and fresh frozen plasma induced a remission, and administration of warfarin has been successful in preventing recurrence of attacks for as long as 8 months without infusion of any plasma components. None of the family members who are heterozygous for protein C deficiency have had thrombotic episodes.

A Registry Study on Treatment of Patients with Acquired Protein C Deficiency with a Protein C Concentrate (Human)

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3816-3816
Author(s):  
Paul Knoebl ◽  
Michael Sasse ◽  
Maria Pia De Carolis ◽  
Jacqueline A Dyck-Jones ◽  
Sandra Ehrle ◽  
...  
Keyword(s):  
Protein C ◽  
Purpura Fulminans ◽  
Protein C Deficiency ◽  
Employment Equity ◽  
Advisory Committees ◽  
Protein C Concentrate ◽  
Concentrate Treatment ◽  
Uremic Syndrome ◽  
Equity Ownership ◽  
The Eu

Abstract The objective of this non-interventional, international registry was to collect and assess data on the indications, safety, and the outcomes of treatment of patients with acquired protein C deficiency with a plasma-derived, human non-activated protein C concentrate (Ceprotin, Baxalta/Shire). Any patient who received treatment with protein C concentrate was eligible for the study; there were no exclusion criteria. This was an observational study with no required predefined visits, medical or laboratory tests, procedures or interventions either at enrollment or during the study. The study was conducted from June 2010 to June 2015. Management of patients was according to the discretion of the investigator. The primary endpoints were the medical diagnoses associated with protein C concentrate treatment, the protein C concentrate treatment regimens (dose, frequency), treatment outcomes and safety information. Here we report the results from 18 patients with severe acquired protein C deficiency (SAPCD - as judged by the treating physician) from 4 European (EU) investigative sites. Protein C concentrate is not currently approved for treatment of acquired protein C deficiency in the EU. The median age at study entry was 1.9 years (range: birth to 73.1 years); 5 [27.8%] patients were <1 month, 4 (22%) from 1 month to <2 years of age, 2 (11.1%) from 2 to <12 years, 2 (11.1%) from 12 to <18 and 5 (27.8%) ≥18 years of age. Eighteen patients were treated with protein C concentrate. At clinical presentation, 15/18 (83.3%) of these patients had sepsis, 2 had necrotizing enterocolitis and 1 had hemolytic uremic syndrome. Thromboembolic disease was present in 8/18 (25.8%) patients, all of whom had purpura fulminans. Of these 8 patients, 2 had disseminated intravascular coagulation (DIC) in addition to other conditions; 1 patient also had arterial thrombosis, and the other patient had macrovascular thrombosis and skin necrosis. The remaining 10 (74.2%) patients had no evidence of thromboembolic disease: of these, 7 patients had sepsis, 2 necrotizing enterocolitis and 1 hemolytic uremic syndrome. Dose level and frequency were variable: the most common dosage was 100 IU/kg and the most common interval between doses was 6 hours. There were no infusions administered for surgical interventions or for prophylaxis. At the time of final analysis 17/18 (94.4%) of patients had at least one follow-up visit. The median duration of study participation was 17.8 months (range 0.4-39.6 months). The mortality rate was 2/18 (11%) patients, including one case of fatal peritonitis, coinciding with protein C concentrate treatment in a 44 day-old, very low birthweight preterm infant and a case of multi-organ failure in a 19 year-old patient. Both deaths were considered not related to protein C concentrate. Ten patients received anticoagulation therapies in addition to protein C including: 4 treatments with antithrombin concentrates, 1 with clopidogrel, 6 with low molecular weight heparin and 7 with unfractionated heparin. Data from historical protein C concentrate treatments which occurred prior to enrollment in the study were collected from 3 patients upon their enrollment into the study (all 3 treatments resulted in halting/reversal of coagulopathy). During the entire study period, 13 adverse events (AEs) were reported in 5 patients with acquired deficiency; among these, 4 were serious adverse events (SAEs). During the period of treatment with protein C concentrate, there were 2 AEs including an SAE of gastroenteritis; neither were considered related AEs. The results of this registry demonstrate that in current clinical practice in the EU, treatment with protein C concentrate was safe with a mortality rate of 11% in patients with acute SAPCD and thrombotic episodes observed in association with sepsis and/or purpura fulminans in this study. Disclosures Knoebl: Baxalta, now part of Shire: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Sasse:Baxter/Baxalta/Shire: Membership on an entity's Board of Directors or advisory committees, Research Funding. De Carolis:Baxalta/Baxter: Other: Baxter Investigator for CEPROTIN Registry. Dyck-Jones:Baxter/Baxalta/Shire: Employment, Equity Ownership. Ehrle:Baxter/Baxalta/Shire: Employment, Equity Ownership. Finnerty:Baxalta/Baxter: Employment. Gelmont:Baxter/Baxalta/Shire: Employment, Equity Ownership. Yel:Baxter/Baxalta/Shire: Employment, Equity Ownership. Loghman-Adham:Baxter/Baxalta/Shire: Employment, Equity Ownership. Fischer:Baxter/Baxalta GmBH: Honoraria, Other: 'Weimarer Sepsis Update 2013'; Baxter/Baxalta GmBH: Consultancy, Other: Co-Authorship for E- Book-Preparing (Importance of the Protein C pathway in coagulation disorders and intensive care medicine / Treatment of congenital and acquired protein C deficiency.

Purpura fulminans in congenital protein C deficiency successfully treated with fresh frozen plasma and thrombomodulin

2017 ◽  
Vol 45 (6) ◽  
pp. e165-e166
Author(s):  
Takuma Hayami ◽  
Akihiko Yamaguchi ◽  
Takeshi Kato ◽  
Toshihiro Tanaka ◽  
Yuka Nishizawa ◽  
...  
Keyword(s):  
Protein C ◽  
Purpura Fulminans ◽  
Fresh Frozen Plasma ◽  
Protein C Deficiency ◽  
Fresh Frozen ◽  
Frozen Plasma ◽  
Congenital Protein C Deficiency

Activated Protein C Concentrate: A New Tool for the Treatment of Acute Thromboembolism in Patients with Congenital Protein C Deficiency

1997 ◽  
Vol 77 (06) ◽  
pp. 1223-1224 ◽  
Author(s):  
Mitsuhiko Sugimoto ◽  
Yoshiyuki Maruhashi ◽  
Atsushi Kobayashi ◽  
Nobuhiro Narita ◽  
Akira Yoshioka
Keyword(s):  
Protein C ◽  
Activated Protein C ◽  
Protein C Deficiency ◽  
Protein C Concentrate ◽  
Congenital Protein C Deficiency
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