Clinical picture and treatment of 2212 patients with common
variable immunodeficiency

J 2014

Clinical picture and treatment of 2212 patients with common variable immunodeficiency

GATHMANN, Benjamin, Nizar MAHLAOUI, Laurence GÉRARD, Eric OKSENHENDLER, Klaus WARNATZ et. al.

Basic information

Original name

Clinical picture and treatment of 2212 patients with common variable immunodeficiency

Authors

GATHMANN, Benjamin (276 Germany), Nizar MAHLAOUI (250 France), Laurence GÉRARD (250 France), Eric OKSENHENDLER (250 France), Klaus WARNATZ (276 Germany), Ilka SCHULZE (276 Germany), Gerhard KINDLE (276 Germany), Taco W. KUIJPERS (528 Netherlands), Rachel T. van BEEM (528 Netherlands), David GUZMAN (840 United States of America), Sarita WORKMAN (826 United Kingdom of Great Britain and Northern Ireland), Pete SOLER-PALACÍN (724 Spain), Javier De GRACIA (724 Spain), Torsten WITTE (276 Germany), Reinhold E. SCHMIDT (276 Germany), Jiří LITZMAN (203 Czech Republic, guarantor, belonging to the institution), Eva HLAVACKOVA (203 Czech Republic), Vojtěch THON (203 Czech Republic, belonging to the institution), Michael BORTE (276 Germany), Stephan BORTE (276 Germany), Dinakantha KUMARARATNE (826 United Kingdom of Great Britain and Northern Ireland), Conleth FEIGHERY (372 Ireland), Hilary LONGHURST (826 United Kingdom of Great Britain and Northern Ireland), Matthew HELBERT (826 United Kingdom of Great Britain and Northern Ireland), Anna SZAFLARSKA (616 Poland), Anna SEDIVA (203 Czech Republic), Bernd H. BELOHRADSKY (276 Germany), Alison JONES (826 United Kingdom of Great Britain and Northern Ireland), Ulrich BAUMANN (276 Germany), Isabelle MEYTS (56 Belgium), Necil KUTUKCULER (792 Turkey), Per WAGSTROM (752 Sweden), Nermeen Mouftah GALAL (818 Egypt), Joachim ROESLER (276 Germany), Evangelia FARMAKI (300 Greece), Natalia ZINOVIEVA (643 Russian Federation), Peter CIZNAR (703 Slovakia), Efimia PAPADOPOULOU-ALATAKI (300 Greece), Kirsten BIENEMANN (276 Germany), Sirje VELBRI (233 Estonia), Zoya PANAHLOO (826 United Kingdom of Great Britain and Northern Ireland) and Bodo GRIMBACHER (276 Germany)

Edition

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, New York, Mosby-Elsevier, 2014, 0091-6749

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30102 Immunology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

Impact factor

Impact factor: 11.476

RIV identification code

RIV/00216224:14110/14:00077279

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1016/j.jaci.2013.12.1077

UT WoS

000338930300016

Keywords in English

Common variable immunodeficiency; immunoglobulin replacement; patient self-reported outcomes; quality of life; primary antibody deficiency; autoimmunity; enteropathy; granulomas; lymphadenopathy; treatment

Abstract

V originále

Background: Common variable immunodeficiency (CVID) is an antibody deficiency with an equal sex distribution and a high variability in clinical presentation. The main features include respiratory tract infections and their associated complications, enteropathy, autoimmunity, and lymphoproliferative disorders. Objective: This study analyzes the clinical presentation, association between clinical features, and differences and effects of immunoglobulin treatment in Europe. Methods: Data on 2212 patients with CVID from 28 medical centers contributing to the European Society for Immunodeficiencies Database were analyzed retrospectively. Results: Early disease onset (< 10 years) was very frequent in our cohort (33.7%), especially in male subjects (39.8%). Male subjects with early-onset CVID were more prone to pneumonia and less prone to other complications suggesting a distinct disease entity. The diagnostic delay of CVID ranges between 4 and 5 years in many countries and is particularly high in subjects with early-onset CVID. Enteropathy, autoimmunity, granulomas, and splenomegaly formed a set of interrelated features, whereas bronchiectasis was not associated with any other clinical feature. Patient survival in this cohort was associated with age at onset and age at diagnosis only. There were different treatment strategies in Europe, with considerable differences in immunoglobulin dosing, ranging from 130 up to 750 mg/kg/mo. Patients with very low trough levels of less than 4 g/L had poor clinical outcomes, whereas higher trough levels were associated with a reduced frequency of serious bacterial infections. Conclusion: Patients with CVID are being managed differently throughout Europe, affecting various outcome measures. Clinically, CVID is a truly variable antibody deficiency syndrome.

Citovat

GATHMANN, Benjamin, Nizar MAHLAOUI, Laurence GÉRARD, Eric OKSENHENDLER, Klaus WARNATZ, Ilka SCHULZE, Gerhard KINDLE, Taco W. KUIJPERS, Rachel T. van BEEM, David GUZMAN, Sarita WORKMAN, Pete SOLER-PALACÍN, Javier De GRACIA, Torsten WITTE, Reinhold E. SCHMIDT, Jiří LITZMAN, Eva HLAVACKOVA, Vojtěch THON, Michael BORTE, Stephan BORTE, Dinakantha KUMARARATNE, Conleth FEIGHERY, Hilary LONGHURST, Matthew HELBERT, Anna SZAFLARSKA, Anna SEDIVA, Bernd H. BELOHRADSKY, Alison JONES, Ulrich BAUMANN, Isabelle MEYTS, Necil KUTUKCULER, Per WAGSTROM, Nermeen Mouftah GALAL, Joachim ROESLER, Evangelia FARMAKI, Natalia ZINOVIEVA, Peter CIZNAR, Efimia PAPADOPOULOU-ALATAKI, Kirsten BIENEMANN, Sirje VELBRI, Zoya PANAHLOO and Bodo GRIMBACHER. Clinical picture and treatment of 2212 patients with common variable immunodeficiency. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY. New York: Mosby-Elsevier, 2014, vol. 134, No 1, p. "116"-"+", 22 pp. ISSN 0091-6749. Available from: https://dx.doi.org/10.1016/j.jaci.2013.12.1077.

@article{1205758,
   author = {Gathmann, Benjamin and Mahlaoui, Nizar and Gérard, Laurence and Oksenhendler, Eric and Warnatz, Klaus and Schulze, Ilka and Kindle, Gerhard and Kuijpers, Taco W. and Beem, Rachel T. van and Guzman, David and Workman, Sarita and SolerandPalacín, Pete and Gracia, Javier De and Witte, Torsten and Schmidt, Reinhold E. and Litzman, Jiří and Hlavackova, Eva and Thon, Vojtěch and Borte, Michael and Borte, Stephan and Kumararatne, Dinakantha and Feighery, Conleth and Longhurst, Hilary and Helbert, Matthew and Szaflarska, Anna and Sediva, Anna and Belohradsky, Bernd H. and Jones, Alison and Baumann, Ulrich and Meyts, Isabelle and Kutukculer, Necil and Wagstrom, Per and Galal, Nermeen Mouftah and Roesler, Joachim and Farmaki, Evangelia and Zinovieva, Natalia and Ciznar, Peter and PapadopoulouandAlataki, Efimia and Bienemann, Kirsten and Velbri, Sirje and Panahloo, Zoya and Grimbacher, Bodo},
   article_location = {New York},
   article_number = {1},
   doi = {http://dx.doi.org/10.1016/j.jaci.2013.12.1077},
   keywords = {Common variable immunodeficiency; immunoglobulin replacement; patient self-reported outcomes; quality of life; primary antibody deficiency; autoimmunity; enteropathy; granulomas; lymphadenopathy; treatment},
   language = {eng},
   issn = {0091-6749},
   journal = {JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY},
   title = {Clinical picture and treatment of 2212 patients with common variable immunodeficiency},
   volume = {134},
   year = {2014}
}

TY  - JOUR
ID  - 1205758
AU  - Gathmann, Benjamin - Mahlaoui, Nizar - Gérard, Laurence - Oksenhendler, Eric - Warnatz, Klaus - Schulze, Ilka - Kindle, Gerhard - Kuijpers, Taco W. - Beem, Rachel T. van - Guzman, David - Workman, Sarita - Soler-Palacín, Pete - Gracia, Javier De - Witte, Torsten - Schmidt, Reinhold E. - Litzman, Jiří - Hlavackova, Eva - Thon, Vojtěch - Borte, Michael - Borte, Stephan - Kumararatne, Dinakantha - Feighery, Conleth - Longhurst, Hilary - Helbert, Matthew - Szaflarska, Anna - Sediva, Anna - Belohradsky, Bernd H. - Jones, Alison - Baumann, Ulrich - Meyts, Isabelle - Kutukculer, Necil - Wagstrom, Per - Galal, Nermeen Mouftah - Roesler, Joachim - Farmaki, Evangelia - Zinovieva, Natalia - Ciznar, Peter - Papadopoulou-Alataki, Efimia - Bienemann, Kirsten - Velbri, Sirje - Panahloo, Zoya - Grimbacher, Bodo
PY  - 2014
TI  - Clinical picture and treatment of 2212 patients with common variable immunodeficiency
JF  - JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
VL  - 134
IS  - 1
SP  - "116"-"+"
EP  - "116"-"+"
PB  - Mosby-Elsevier
SN  - 00916749
KW  - Common variable immunodeficiency
KW  - immunoglobulin replacement
KW  - patient self-reported outcomes
KW  - quality of life
KW  - primary antibody deficiency
KW  - autoimmunity
KW  - enteropathy
KW  - granulomas
KW  - lymphadenopathy
KW  - treatment
N2  - Background: Common variable immunodeficiency (CVID) is an antibody deficiency with an equal sex distribution and a high variability in clinical presentation. The main features include respiratory tract infections and their associated complications, enteropathy, autoimmunity, and lymphoproliferative disorders. Objective: This study analyzes the clinical presentation, association between clinical features, and differences and effects of immunoglobulin treatment in Europe. Methods: Data on 2212 patients with CVID from 28 medical centers contributing to the European Society for Immunodeficiencies Database were analyzed retrospectively. Results: Early disease onset (< 10 years) was very frequent in our cohort (33.7%), especially in male subjects (39.8%). Male subjects with early-onset CVID were more prone to pneumonia and less prone to other complications suggesting a distinct disease entity. The diagnostic delay of CVID ranges between 4 and 5 years in many countries and is particularly high in subjects with early-onset CVID. Enteropathy, autoimmunity, granulomas, and splenomegaly formed a set of interrelated features, whereas bronchiectasis was not associated with any other clinical feature. Patient survival in this cohort was associated with age at onset and age at diagnosis only. There were different treatment strategies in Europe, with considerable differences in immunoglobulin dosing, ranging from 130 up to 750 mg/kg/mo. Patients with very low trough levels of less than 4 g/L had poor clinical outcomes, whereas higher trough levels were associated with a reduced frequency of serious bacterial infections. Conclusion: Patients with CVID are being managed differently throughout Europe, affecting various outcome measures. Clinically, CVID is a truly variable antibody deficiency syndrome.
ER  -

GATHMANN, Benjamin, Nizar MAHLAOUI, Laurence GÉRARD, Eric OKSENHENDLER, Klaus WARNATZ, Ilka SCHULZE, Gerhard KINDLE, Taco W. KUIJPERS, Rachel T. van BEEM, David GUZMAN, Sarita WORKMAN, Pete SOLER-PALACÍN, Javier De GRACIA, Torsten WITTE, Reinhold E. SCHMIDT, Jiří LITZMAN, Eva HLAVACKOVA, Vojtěch THON, Michael BORTE, Stephan BORTE, Dinakantha KUMARARATNE, Conleth FEIGHERY, Hilary LONGHURST, Matthew HELBERT, Anna SZAFLARSKA, Anna SEDIVA, Bernd H. BELOHRADSKY, Alison JONES, Ulrich BAUMANN, Isabelle MEYTS, Necil KUTUKCULER, Per WAGSTROM, Nermeen Mouftah GALAL, Joachim ROESLER, Evangelia FARMAKI, Natalia ZINOVIEVA, Peter CIZNAR, Efimia PAPADOPOULOU-ALATAKI, Kirsten BIENEMANN, Sirje VELBRI, Zoya PANAHLOO and Bodo GRIMBACHER. Clinical picture and treatment of 2212 patients with common variable immunodeficiency. \textit{JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY}. New York: Mosby-Elsevier, 2014, vol.~134, No~1, p.~''116''-''+'', 22 pp. ISSN~0091-6749. Available from: https://dx.doi.org/10.1016/j.jaci.2013.12.1077.

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