Abstract:
: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 28 518 patient-years (PY) (PNH, 21 016 PY; aHUS, 7502 PY). Seventy-six cases of meningococcal infection were reported (0·25/100 PY), including eight fatal PNH cases (0·03/100 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 (11·8%); bacteraemia, sepsis and septic shock (11·1%); urinary tract infection (4·1%); staphylococcal infection (2·6%); and viral infection (2·5%). 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 (≈0·6/100 PY) and haematological malignancies (≈0·74/100 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.
journal_name
Br J Haematoljournal_title
British journal of haematologyauthors
Socié G,Caby-Tosi MP,Marantz JL,Cole A,Bedrosian CL,Gasteyger C,Mujeebuddin A,Hillmen P,Vande Walle J,Haller Hdoi
10.1111/bjh.15790subject
Has Abstractpub_date
2019-04-01 00:00:00pages
297-310issue
2eissn
0007-1048issn
1365-2141journal_volume
185pub_type
杂志文章abstract::Mature (non-anaplastic) T-cell and natural killer (NK)-lymphomas rarely occur in children or adolescents. Due to the low incidence and heterogeneity, information regarding the aetiology, physiopathology and genetics of paediatric mature (non-anaplastic) T/NK-cell lymphoma is lacking. In addition, standard treatments h...
journal_title:British journal of haematology
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journal_title:British journal of haematology
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abstract::Deficiency in DNA repair capability is considered to be responsible for oncogenesis. Hereditary and sporadic cancers in various tissues have been reported to have mutations at the DNA repair genes. In this study we analysed two excision repair genes (ERCC1 and XPCC) and two mismatch repair genes (hMSH2 and hMTH1) in t...
journal_title:British journal of haematology
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abstract::Blood donor screening for antibody to hepatitis B core antigen (anti-HBc) implemented in some countries as a surrogate marker for non-A, non-B hepatitis has been superseded by anti-HCV screening. To assess the value of anti-HBc screening for the detection of hepatitis B surface antigen-negative blood donations that mi...
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doi:10.1111/j.1365-2141.1978.tb03640.x
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abstract::Diamond-Blackfan anaemia (DBA) is a constitutional pure red cell aplasia presenting in early childhood. In some patients, neutropenia and/or thrombocytopenia have also been observed during the course of the disease. We have followed 28 patients with steroid-refractory DBA for up to 13 years with serial peripheral bloo...
journal_title:British journal of haematology
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doi:10.1046/j.1365-2141.2000.01796.x
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journal_title:British journal of haematology
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pub_type: 杂志文章
doi:10.1111/j.1365-2141.2007.06920.x
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journal_title:British journal of haematology
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doi:10.1111/j.1365-2141.1993.tb03219.x
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journal_title:British journal of haematology
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journal_title:British journal of haematology
pub_type: 杂志文章
doi:10.1111/j.1365-2141.1979.tb03718.x
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pub_type: 杂志文章,评审
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journal_title:British journal of haematology
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journal_title:British journal of haematology
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journal_title:British journal of haematology
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doi:10.1046/j.1365-2141.1998.00763.x
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journal_title:British journal of haematology
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