Eculizumab in paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome : 10‐year pharmacovigilance analysis

Autor: Alexander Cole, Marie-Pierre Caby-Tosi, Camille L. Bedrosian, Jing L. Marantz, Hermann Haller, Arshad Mujeebuddin, Peter Hillmen, Gérard Socié, Johan Vande Walle, Christoph Gasteyger
Jazyk: angličtina
Rok vydání: 2019
Předmět:
Male
Databases
Factual
IMPACT
Hemoglobinuria
Paroxysmal
DISEASE
Hemoglobins
Pharmacovigilance
0302 clinical medicine
CLINICAL CHARACTERISTICS
Pregnancy
Medicine and Health Sciences
Medicine
Child
Fatigue
Atypical Hemolytic Uremic Syndrome
Hematology
Pregnancy Outcome
Middle Aged
Eculizumab
PREGNANT-WOMAN
Child
Preschool
030220 oncology & carcinogenesis
Female
eculizumab
COMPLEMENT INHIBITOR ECULIZUMAB
Research Paper
medicine.drug
Adult
safety
medicine.medical_specialty
Adolescent
Fever
Urinary system
UNITED-STATES
Opportunistic Infections
paroxysmal nocturnal haemoglobinuria
Antibodies
Monoclonal
Humanized
Sepsis
Young Adult
03 medical and health sciences
Internal medicine
atypical haemolytic uraemic syndrome
WHOLE-BLOOD
Humans
Red Cells and Iron
Aged
business.industry
Septic shock
MUTATIONS
Infant
Newborn
Infant
medicine.disease
EFFICACY
Meningococcal Infections
Pregnancy Complications
Pneumonia
Complement Inactivating Agents
pharmacovigilance
Paroxysmal nocturnal haemoglobinuria
business
030215 immunology
Zdroj: BRITISH JOURNAL OF HAEMATOLOGY
British Journal of Haematology
ISSN: 0007-1048
1365-2141
Popis: Eculizumab is the first and only medication approved for paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) treatment. However, eculizumab safety based on long-term pharmacovigilance is unknown. This analysis summarises safety data collected from spontaneous and solicited sources from 16 March 2007 through 1 October 2016. Cumulative exposure to eculizumab was 28518 patient-years (PY) (PNH, 21016 PY; aHUS, 7502 PY). Seventy-six cases of meningococcal infection were reported (025/100 PY), including eight fatal PNH cases (003/100 PY). Susceptibility to meningococcal infections remained the key risk in patients receiving eculizumab. The meningococcal infection rate decreased over time; related mortality remained steady. The most commonly reported serious nonmeningococcal infections were pneumonia (118%); bacteraemia, sepsis and septic shock (111%); urinary tract infection (41%); staphylococcal infection (26%); and viral infection (25%). There were 434 reported cases of eculizumab exposure in pregnant women; of 260 cases with known outcomes, 70% resulted in live births. Reporting rates for solid tumours (approximate to 06/100 PY) and haematological malignancies (approximate to 074/100 PY) remained stable over time. No new safety signals affecting the eculizumab benefit-risk profile were identified. Continued awareness and implementation of risk mitigation protocols are essential to minimise risk of meningococcal and other Neisseria infections in patients receiving eculizumab.
Databáze: OpenAIRE
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