FranceCoag: a 22-year prospective follow-up of the national French cohort of patients with inherited bleeding disorders

Doncarli, Alexandra; Demiguel, Virginie; Canu, Irina Guseva; Goulet, V.; Bayart, Sophie; Calvez, Thierry; Castet, Sabine; Dalibard, Vincent; Yohan, Demay; Frotscher, Birgit; Goudemand, Jenny; Lambert, Thierry; Milien, Vanessa; Oudot, Caroline; Sannié, Thomas; Chambost, Hérvè

doi:10.1007/s10654-018-0468-7

Cited by 19 publications

(18 citation statements)

References 12 publications

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“…Our cohort involved the entire set of French patients with FXIII deficiency <10 iu/dl registered in the FranceCoag Network (Doncarli et al , )), which relies on comprehensive data concerning haemophilia and RDBs from 40 centres. Our study is one of the largest and most homogeneous FXIII deficiency cases studied to date regarding disease definition, severity and families included ( n = 31).…”

Section: Discussionmentioning

confidence: 99%

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Congenital factor XIII deficiency: comprehensive overview of the FranceCoag cohort

Bouttefroy

Chambost

Milien³

et al. 2019

Br J Haematol

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This FranceCoag network study assessed 33 patients with congenital factor XIII (FXIII) deficiency presenting FXIII levels <10 iu/dl. Diagnosis was based on abnormal bleeding in 29 patients, a positive family history in 2, recurrent miscarriages in 1 and was fortuitous in 1. Eighteen patients (62Á1%) presented life-threatening umbilical or intracranial haemorrhages (ICH). Seven of the 15 patients who experienced ICH were diagnosed but untreated, including 3 with secondary neurological sequelae. All pregnancies without prophylaxis (26/26) led to miscarriages versus 3/16 with prophylaxis. In patients exhibiting FXIII levels <10 iu/dl, prophylaxis could be discussed at diagnosis and at pregnancy. Further controlled prospective studies are needed.

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Section: Discussionmentioning

confidence: 99%

“…This retrospective study, conducted from October 2017 to July 2018, was based on the FranceCoag Network (Doncarli et al , ), a national prospective cohort of patients with inherited coagulation factor deficiencies. The patients, followed‐up in a haemophilia centre, presented FXIII activity levels <10 iu/dl.…”

Section: Methodsmentioning

confidence: 99%

Congenital factor XIII deficiency: comprehensive overview of the FranceCoag cohort

Bouttefroy

Chambost

Milien³

et al. 2019

Br J Haematol

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Section: Intramuscular Vaccination Of Haemophiliacs: Is It Really a Rmentioning

confidence: 99%

“…The population of the present study was composed of patients included in the national haemophiliac cohort (FranceCoag) by the haemophilia care centres of Bordeaux, Lille, Lyon and Marseille; FranceCoag is a national prospective cohort of patients with inherited coagulation factor deficiencies. 9 We included all haemophilic patients, with factor VIII or IX <2%,The exclusion criteria were a diagnosis of haemophilia made before 2 months of age (age of first vaccine administration in France) and/ or the absence of intramuscular vaccination at the time of diagnosis.We thereby obtained a homogenous population of severe or moderate haemophiliacs who had received at least one intramuscular vaccination (according to the French recommendations) before the diagnosis of haemophilia. No specific precautions (such as prolonged compression) were used for these children.…”

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confidence: 99%

Intramuscular vaccination of haemophiliacs: Is it really a risk for bleeding?

Hochart¹,

Falaise²,

Huguenin

et al. 2019

Haemophilia

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In patients with haemophilia, it is consensual that all routine vaccinations should be given at the appropriate time following national guidelines. 1,2 However, whether these should be administered using the intramuscular or subcutaneous route is still under debate as there is a challenging risk-benefit balance in this specific population. In France, the consensus is to perform all vaccinations by the subcutaneous route for all haemophiliacs. The risk associated with intramuscular injection is the development of intramuscular bleeding leading to factor exposure and potential inhibitor risk, 1,2 whereas subcutaneous injection increases the risk of local side effects (oedema, erythema, itching and granuloma formation) in particular for adjuvant-containing vaccines. [3][4][5] In addition, there is ongoing debate regarding vaccine immunogenicity and tolerability with this alternate route of administration; for example, immunogenicity appears to be decreased in case of subcutaneous injection as reflected by significantly lower seroconversion rates and more rapid decay of antibody response. [3][4][5] This may be in relation to the poor vascularity of this localization that may result in slow mobilization and processing of antigen presentation. [3][4][5][6] Recent data may modify this risk-benefit balance. Last year, the European Pediatric Network for Haemophilia Management (PedNet) group found no association between vaccinations given shortly before or after FVIII exposure (48 hours before and 24 hours after) and inhibitor development in previously untreated patients (before 75 EDs); in this study, it is of note that among 375 children with severe haemophilia, the route of administration was intramuscular for 18.9% of patients, subcutaneously for 74.0%, and unknown for 6.9%. 7 The occurrence of inhibitor remains the primary concern of physicians when haemophilia is diagnosed, in particular, if severe or moderate. The choice of the subcutaneous route may thus limit haemorrhagic complications and early use of antihaemophilic treatment. However, we know that some countries (such as France) are currently facing vaccine hesitancy or vaccine refusal leading to a resurgence of pertussis and measles, 8 but also to insufficient herd immunity to protect haemophilic patients with potentially lower seroconversion rates after subcutaneous vaccination.We, therefore, decided to evaluate the development of intramuscular haematoma requiring factor infusion in case of intramuscular vaccination in severe and moderate (<2%) haemophilic patients. The population of the present study was composed of patients included in the national haemophiliac cohort (FranceCoag) by the haemophilia care centres of Bordeaux, Lille, Lyon and Marseille; FranceCoag is a national prospective cohort of patients with inherited coagulation factor deficiencies. 9 We included all haemophilic patients, with factor VIII or IX <2%,The exclusion criteria were a diagnosis of haemophilia made before 2 months of age (age of first vaccine administration in France) and/ or t...

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“…In this paper, the term Registry will be used with the same meaning as the terms ‘Register’ and, respectively, ‘Database’. There are several types of registries on different levels collecting data on haemophilia which may be based on the treatment centre cohort, the country cohort, such as the ones existing in the UK and France (http://www.ukhcdo.org/; http://www.francecoag.org) European or transregional registries such as the PedNet (http://pednet.eu; http://web.euhass.org), or global ones such as the World Federation of Hemophilia (WFH) World Bleeding Disorders Registry (https://www.wfh.org/en/wbdr). In addition, there are all the product‐based data collections from clinical trials that may be used for similar purposes.…”

Section: The Types Of Registriesmentioning

confidence: 99%

Registries and databases—A European perspective

Ljung

2020

Haemophilia

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Registries will enable cohort studies to be performed, which are usually considered to be the best quality of observational studies. The quality of data of registries can be increased if is it possible to merge results ('crosstalk') between registries. A prerequisite for that is an agreed uniform core set of data to be collected and uniform definitions on the items to be collected. This paper discusses problems and barriers with existing registries and provides recommendations from an EMA workshop (European Medicines Agency), for core common data sets and how to secure the quality of data collected. The PedNet registry including >2200 children with haemophilia is presented as an example of a registry/cohort study. K E Y W O R D Sfactor IX, factor VIII, haemophilia, register

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FranceCoag: a 22-year prospective follow-up of the national French cohort of patients with inherited bleeding disorders

Cited by 19 publications

References 12 publications

Congenital factor XIII deficiency: comprehensive overview of the FranceCoag cohort

Congenital factor XIII deficiency: comprehensive overview of the FranceCoag cohort

Intramuscular vaccination of haemophiliacs: Is it really a risk for bleeding?

Registries and databases—A European perspective

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