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.

Základní údaje

Originální název

Clinical picture and treatment of 2212 patients with common variable immunodeficiency

Autoři

GATHMANN, Benjamin (276 Německo), Nizar MAHLAOUI (250 Francie), Laurence GÉRARD (250 Francie), Eric OKSENHENDLER (250 Francie), Klaus WARNATZ (276 Německo), Ilka SCHULZE (276 Německo), Gerhard KINDLE (276 Německo), Taco W. KUIJPERS (528 Nizozemské království), Rachel T. van BEEM (528 Nizozemské království), David GUZMAN (840 Spojené státy), Sarita WORKMAN (826 Velká Británie a Severní Irsko), Pete SOLER-PALACÍN (724 Španělsko), Javier De GRACIA (724 Španělsko), Torsten WITTE (276 Německo), Reinhold E. SCHMIDT (276 Německo), Jiří LITZMAN (203 Česká republika, garant, domácí), Eva HLAVACKOVA (203 Česká republika), Vojtěch THON (203 Česká republika, domácí), Michael BORTE (276 Německo), Stephan BORTE (276 Německo), Dinakantha KUMARARATNE (826 Velká Británie a Severní Irsko), Conleth FEIGHERY (372 Irsko), Hilary LONGHURST (826 Velká Británie a Severní Irsko), Matthew HELBERT (826 Velká Británie a Severní Irsko), Anna SZAFLARSKA (616 Polsko), Anna SEDIVA (203 Česká republika), Bernd H. BELOHRADSKY (276 Německo), Alison JONES (826 Velká Británie a Severní Irsko), Ulrich BAUMANN (276 Německo), Isabelle MEYTS (56 Belgie), Necil KUTUKCULER (792 Turecko), Per WAGSTROM (752 Švédsko), Nermeen Mouftah GALAL (818 Egypt), Joachim ROESLER (276 Německo), Evangelia FARMAKI (300 Řecko), Natalia ZINOVIEVA (643 Rusko), Peter CIZNAR (703 Slovensko), Efimia PAPADOPOULOU-ALATAKI (300 Řecko), Kirsten BIENEMANN (276 Německo), Sirje VELBRI (233 Estonsko), Zoya PANAHLOO (826 Velká Británie a Severní Irsko) a Bodo GRIMBACHER (276 Německo)

Vydání

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

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30102 Immunology

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Impakt faktor

Impact factor: 11.476

Kód RIV

RIV/00216224:14110/14:00077279

Organizační jednotka

Lékařská fakulta

DOI

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

UT WoS

000338930300016

Klíčová slova anglicky

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

Štítky

EL OK

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 24. 4. 2015 13:55, Soňa Böhmová

Anotace

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 a Bodo GRIMBACHER. Clinical picture and treatment of 2212 patients with common variable immunodeficiency. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY. New York: Mosby-Elsevier, 2014, roč. 134, č. 1, s. "116"-"+", 22 s. ISSN 0091-6749. Dostupné z: 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 a 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, roč.~134, č.~1, s.~''116''-''+'', 22 s. ISSN~0091-6749. Dostupné z: https://dx.doi.org/10.1016/j.jaci.2013.12.1077.

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