The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data
Abstract Background Common variable immunodeficiency disorders (CVID) are a group of rare innate disorders characterized by specific antibody deficiency and increased rates of infections, comorbidities and mortality. The burden of CVID in Europe has not been previously estimated. We performed a retr...
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doaj-04c8e87da665449f9113be8c77916d7d2020-11-25T02:15:41ZengBMCOrphanet Journal of Rare Diseases1750-11722018-11-0113111710.1186/s13023-018-0941-0The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry dataIrina Odnoletkova0Gerhard Kindle1Isabella Quinti2Bodo Grimbacher3Viviane Knerr4Benjamin Gathmann5Stephan Ehl6Nizar Mahlaoui7Philippe Van Wilder8Kris Bogaerts9Esther de Vries10in collaboration with the Plasma Protein Therapeutics Association (PPTA) TaskforcePlasma Protein Therapeutics AssociationThe ESID Registry Working PartyDepartment of Molecular Medicine, Sapienza University of RomeCenter for Chronic Immunodeficiency, Medical Center – University of Freiburg, Faculty of Medicine, University of FreiburgThe ESID Registry Working PartyThe ESID Registry Working PartyThe ESID Registry Working PartyFrench National Reference Center for Primary Immune Deficiencies (CEREDIH) and Pediatric Immuno-Haematology and Rheumatology Unit Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de ParisCentre de recherche en Economie de la Santé, Gestion des Institutions de Soins et Sciences Infirmières, Ecole de Santé Publique, University of Brussels (ULB)Interuniversity Institute for Biostatistics and Statistical Bioinformatics (I-BioStat), KU Leuven – University of Leuven, I-BioStatDepartment Tranzo, Tilburg UniversityAbstract Background Common variable immunodeficiency disorders (CVID) are a group of rare innate disorders characterized by specific antibody deficiency and increased rates of infections, comorbidities and mortality. The burden of CVID in Europe has not been previously estimated. We performed a retrospective analysis of the European Society for Immunodeficiencies (ESID) registry data on the subset of patients classified by their immunologist as CVID and treated between 2004 and 2014. The registered deaths and comorbidities were used to calculate the annual average age-standardized rates of Years of Life Lost to premature death (YLL), Years Lost to Disability (YLD) and Disability Adjusted Life Years (DALY=YLL + YLD). These outcomes were expressed as a rate per 105 of the CVID cohort (the individual disease burden), and of the general population (the societal disease burden). Results Data of 2700 patients from 23 countries were analysed. Annual comorbidity rates: bronchiectasis, 21.9%; autoimmunity, 23.2%; digestive disorders, 15.6%; solid cancers, 5.5%; lymphoma, 3.8%, exceeded the prevalence in the general population by a factor of 34.0, 7.6, 8.1, 2.4 and 32.6, respectively. The comorbidities of CVID caused 8722 (6069; 12,363) YLD/105 in this cohort, whereas 44% of disability burden was attributable to infections and bronchiectasis. The total individual burden of CVID was 36,785 (33,078, 41,380) DALY/105. With estimated CVID prevalence of ~ 1/ 25,000, the societal burden of CVID ensued 1.5 (1.3, 1.7) DALY/105 of the general population. In exploratory analysis, increased mortality was associated with solid tumor, HR (95% CI): 2.69 (1.10; 6.57) p = 0.030, lymphoma: 5.48 (2.36; 12.71) p < .0001 and granulomatous-lymphocytic interstitial lung disease: 4.85 (1.63; 14.39) p = 0.005. Diagnostic delay (median: 4 years) was associated with a higher risk of death: 1.04 (1.02; 1.06) p = .0003, bronchiectasis: 1.03 (1.01; 1.04) p = .0001, solid tumor: 1.08 (1.04; 1.11) p < .0001 and enteropathy: 1.02 (1.00; 1.05) p = .0447 and stayed unchanged over four decades (p = .228). Conclusions While the societal burden of CVID may seem moderate, it is severe to the individual patient. Delay in CVID diagnosis may constitute a modifiable risk factor of serious comorbidities and death but showed no improvement. Tools supporting timely CVID diagnosis should be developed with high priority.http://link.springer.com/article/10.1186/s13023-018-0941-0Primary immunodeficiencyPrimary antibody deficiencyCommon variable immunodeficiencyBurden of diseaseDALYHealth economics |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Irina Odnoletkova Gerhard Kindle Isabella Quinti Bodo Grimbacher Viviane Knerr Benjamin Gathmann Stephan Ehl Nizar Mahlaoui Philippe Van Wilder Kris Bogaerts Esther de Vries in collaboration with the Plasma Protein Therapeutics Association (PPTA) Taskforce |
spellingShingle |
Irina Odnoletkova Gerhard Kindle Isabella Quinti Bodo Grimbacher Viviane Knerr Benjamin Gathmann Stephan Ehl Nizar Mahlaoui Philippe Van Wilder Kris Bogaerts Esther de Vries in collaboration with the Plasma Protein Therapeutics Association (PPTA) Taskforce The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data Orphanet Journal of Rare Diseases Primary immunodeficiency Primary antibody deficiency Common variable immunodeficiency Burden of disease DALY Health economics |
author_facet |
Irina Odnoletkova Gerhard Kindle Isabella Quinti Bodo Grimbacher Viviane Knerr Benjamin Gathmann Stephan Ehl Nizar Mahlaoui Philippe Van Wilder Kris Bogaerts Esther de Vries in collaboration with the Plasma Protein Therapeutics Association (PPTA) Taskforce |
author_sort |
Irina Odnoletkova |
title |
The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data |
title_short |
The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data |
title_full |
The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data |
title_fullStr |
The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data |
title_full_unstemmed |
The burden of common variable immunodeficiency disorders: a retrospective analysis of the European Society for Immunodeficiency (ESID) registry data |
title_sort |
burden of common variable immunodeficiency disorders: a retrospective analysis of the european society for immunodeficiency (esid) registry data |
publisher |
BMC |
series |
Orphanet Journal of Rare Diseases |
issn |
1750-1172 |
publishDate |
2018-11-01 |
description |
Abstract Background Common variable immunodeficiency disorders (CVID) are a group of rare innate disorders characterized by specific antibody deficiency and increased rates of infections, comorbidities and mortality. The burden of CVID in Europe has not been previously estimated. We performed a retrospective analysis of the European Society for Immunodeficiencies (ESID) registry data on the subset of patients classified by their immunologist as CVID and treated between 2004 and 2014. The registered deaths and comorbidities were used to calculate the annual average age-standardized rates of Years of Life Lost to premature death (YLL), Years Lost to Disability (YLD) and Disability Adjusted Life Years (DALY=YLL + YLD). These outcomes were expressed as a rate per 105 of the CVID cohort (the individual disease burden), and of the general population (the societal disease burden). Results Data of 2700 patients from 23 countries were analysed. Annual comorbidity rates: bronchiectasis, 21.9%; autoimmunity, 23.2%; digestive disorders, 15.6%; solid cancers, 5.5%; lymphoma, 3.8%, exceeded the prevalence in the general population by a factor of 34.0, 7.6, 8.1, 2.4 and 32.6, respectively. The comorbidities of CVID caused 8722 (6069; 12,363) YLD/105 in this cohort, whereas 44% of disability burden was attributable to infections and bronchiectasis. The total individual burden of CVID was 36,785 (33,078, 41,380) DALY/105. With estimated CVID prevalence of ~ 1/ 25,000, the societal burden of CVID ensued 1.5 (1.3, 1.7) DALY/105 of the general population. In exploratory analysis, increased mortality was associated with solid tumor, HR (95% CI): 2.69 (1.10; 6.57) p = 0.030, lymphoma: 5.48 (2.36; 12.71) p < .0001 and granulomatous-lymphocytic interstitial lung disease: 4.85 (1.63; 14.39) p = 0.005. Diagnostic delay (median: 4 years) was associated with a higher risk of death: 1.04 (1.02; 1.06) p = .0003, bronchiectasis: 1.03 (1.01; 1.04) p = .0001, solid tumor: 1.08 (1.04; 1.11) p < .0001 and enteropathy: 1.02 (1.00; 1.05) p = .0447 and stayed unchanged over four decades (p = .228). Conclusions While the societal burden of CVID may seem moderate, it is severe to the individual patient. Delay in CVID diagnosis may constitute a modifiable risk factor of serious comorbidities and death but showed no improvement. Tools supporting timely CVID diagnosis should be developed with high priority. |
topic |
Primary immunodeficiency Primary antibody deficiency Common variable immunodeficiency Burden of disease DALY Health economics |
url |
http://link.springer.com/article/10.1186/s13023-018-0941-0 |
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