Search: WFRF:(Hauck Jennifer) >
Therapeutic options...
Therapeutic options for CTLA-4 insufficiency
-
- Egg, David (author)
- Albert Ludwig Univ Freiburg, Germany
-
- Rump, Ina Caroline (author)
- Albert Ludwig Univ Freiburg, Germany
-
- Mitsuiki, Noriko (author)
- Albert Ludwig Univ Freiburg, Germany
-
show more...
-
- Rojas-Restrepo, Jessica (author)
- Albert Ludwig Univ Freiburg, Germany
-
- Maccari, Maria-Elena (author)
- Albert Ludwig Univ Freiburg, Germany; Albert Ludwig Univ Freiburg, Germany
-
- Schwab, Charlotte (author)
- Albert Ludwig Univ Freiburg, Germany
-
- Gabrysch, Annemarie (author)
- Albert Ludwig Univ Freiburg, Germany
-
- Warnatz, Klaus (author)
- Albert Ludwig Univ Freiburg, Germany; Albert Ludwig Univ Freiburg, Germany
-
- Goldacker, Sigune (author)
- Albert Ludwig Univ Freiburg, Germany; Albert Ludwig Univ Freiburg, Germany
-
- Patino, Virginia (author)
- Amer Insurance, Uruguay
-
- Wolff, Daniel (author)
- Univ Hosp Regensburg, Germany
-
- Okada, Satoshi (author)
- Hiroshima Univ, Japan
-
- Hayakawa, Seiichi (author)
- Hiroshima Univ, Japan
-
- Shikama, Yoshiaki (author)
- Kanagawa Childrens Med Ctr, Japan
-
- Kanda, Kenji (author)
- Hikone Municipal Hosp, Japan
-
- Imai, Kohsuke (author)
- Tokyo Med & Dent Univ, Japan
-
- Sotomatsu, Manabu (author)
- Gunma Childrens Med Ctr, Japan
-
- Kuwashima, Makoto (author)
- Kiryu Kosei Gen Hosp, Japan
-
- Kamiya, Takahiro (author)
- Tokyo Med & Dent Univ, Japan
-
- Morio, Tomohiro (author)
- Tokyo Med & Dent Univ, Japan
-
- Matsumoto, Kazuaki (author)
- Gunma Childrens Med Ctr, Japan
-
- Mori, Takeshi (author)
- Hyogo Prefectural Kobe Childrens Hosp, Japan
-
- Yoshimoto, Yuri (author)
- Ctr Hosp Natl Ctr Global Hlth & Med, Japan
-
- Dybedal, Ingunn (author)
- Oslo Univ Hosp, Norway
-
- Kanariou, Maria (author)
- Aghia Sophia Childrens Hosp, Greece
-
- Kucuk, Zeynep Yesim (author)
- Childrens Hosp Med Ctr, OH USA
-
- Chapdelaine, Hugo (author)
- Montreal Clin Res Inst, Canada
-
- Petruzelkova, Lenka (author)
- Charles Univ Prague, Czech Republic
-
- Lorenz, Hanns-Martin (author)
- Heidelberg Univ, Germany
-
- Sullivan, Kathleen E. (author)
- Univ Penn, PA 19104 USA
-
- Heimall, Jennifer (author)
- Univ Penn, PA 19104 USA
-
- Moutschen, Michel (author)
- Univ Hosp Liege, Belgium
-
- Litzman, Jiri (author)
- Masaryk Univ, Czech Republic; St Annes Univ Hosp, Czech Republic
-
- Recher, Mike (author)
- Univ Hosp, Switzerland; Univ Hosp, Switzerland
-
- Albert, Michael H. (author)
- Ludwig Maximilians Univ Munchen, Germany
-
- Hauck, Fabian (author)
- Ludwig Maximilians Univ Munchen, Germany
-
- Seneviratne, Suranjith (author)
- UCL, England
-
- Schmid, Jana Pachlopnik (author)
- Univ Zurich, Switzerland
-
- Kolios, Antonios (author)
- Univ Zurich, Switzerland
-
- Unglik, Gary (author)
- Royal Melbourne Hosp, Australia
-
- Klemann, Christian (author)
- Albert Ludwig Univ Freiburg, Germany; Albert Ludwig Univ Freiburg, Germany
-
- Snapper, Scott (author)
- Harvard Med Sch, MA USA
-
- Giulino-Roth, Lisa (author)
- Weill Cornell Med, NY USA
-
- Svaton, Michael (author)
- Charles Univ Prague, Czech Republic; Univ Hosp Motol, Czech Republic
-
- Platt, Craig D. (author)
- Harvard Med Sch, MA 02115 USA; Harvard Med Sch, MA 02115 USA
-
- Hambleton, Sophie (author)
- Newcastle Upon Tyne Hosp NHS Fdn Trust, England; Newcastle Univ, England
-
- Neth, Olaf (author)
- Hosp Virgen del Roc, Spain
-
- Gosse, Geraldine (author)
- Univ Montreal, Canada
-
- Reinsch, Steffen (author)
- Jena Univ Hosp, Germany
-
- Holzinger, Dirk (author)
- Univ Duisburg Essen, Germany
-
- Kim, Yae-Jean (author)
- Sungkyunkwan Univ, South Korea
-
- Bakhtiar, Shahrzad (author)
- Goethe Univ Frankfurt, Germany
-
- Atschekzei, Faranaz (author)
- Hannover Med Sch, Germany
-
- Schmidt, Reinhold (author)
- Hannover Med Sch, Germany
-
- Sogkas, Georgios (author)
- Hannover Med Sch, Germany
-
- Chandrakasan, Shanmuganathan (author)
- Emory Univ, GA USA
-
- Rae, William (author)
- Univ Hosp Southampton NHSFT, England; Univ Hosp Southampton NHSFT, England
-
- Derfalvi, Beata (author)
- IWK Hlth Ctr, Canada; Dalhousie Univ, Canada
-
- Marquart, Hanne Vibeke (author)
- Univ Copenhagen, Denmark
-
- Ozen, Ahmet (author)
- Marmara Univ, Turkey
-
- Kiykim, Ayca (author)
- Marmara Univ, Turkey
-
- Karakoc-Aydiner, Elif (author)
- Marmara Univ, Turkey
-
- Kralickova, Pavlina (author)
- Charles Univ Prague, Czech Republic
-
- de Bree, Godelieve (author)
- Univ Amsterdam, Netherlands
-
- Kiritsi, Dimitra (author)
- Albert Ludwig Univ Freiburg, Germany
-
- Seidel, Markus G. (author)
- Med Univ Graz, Austria
-
- Kobbe, Robin (author)
- Univ Med Ctr Hamburg Eppendorf, Germany
-
- Dantzer, Jennifer (author)
- Johns Hopkins Univ, MD USA
-
- Alsina, Laia (author)
- Hosp St Joan de Deu, Spain; Inst Recerca St Joan de Deu, Spain; Univ Barcelona, Spain
-
- Armangue, Thais (author)
- Univ Barcelona, Spain
-
- Lougaris, Vassilios (author)
- Univ Barcelona, Spain
-
- Agyeman, Philipp (author)
- Univ Brescia, Italy; Univ Brescia, Italy
-
- Nystrom, Sofia (author)
- Univ Bern, Switzerland
-
- Buchbinder, David (author)
- Linköpings universitet,Institutionen för biomedicinska och kliniska vetenskaper,Medicinska fakulteten,Region Östergötland, Klinisk immunologi och transfusionsmedicin
-
- Arkwright, Peter D. (author)
- Childrens Hosp Orange Cty, CA USA
-
- Grimbacher, Bodo (author)
- Albert Ludwig Univ Freiburg, Germany; Albert Ludwig Univ Freiburg, Germany; Albert Ludwig Univ Freiburg, Germany; UCL, England; Univ Manchester, England; German Ctr Infect Res, Germany; Hannover Med Sch, Germany
-
show less...
-
(creator_code:org_t)
- MOSBY-ELSEVIER, 2022
- 2022
- English.
-
In: Journal of Allergy and Clinical Immunology. - : MOSBY-ELSEVIER. - 0091-6749 .- 1097-6825. ; 149:2, s. 736-746
- Related links:
-
https://urn.kb.se/re...
-
show more...
-
https://doi.org/10.1...
-
show less...
Abstract
Subject headings
Close
- Background: Heterozygous germline mutations in cytotoxic T lymphocyte-associated antigen-4 (CTLA4) impair the immunomodulatory function of regulatory T cells. Affected individuals are prone to life-threatening autoimmune and lymphoproliferative complications. A number of therapeutic options are currently being used with variable effectiveness. Objective: Our aim was to characterize the responsiveness of patients with CTLA-4 insufficiency to specific therapies and provide recommendations for the diagnostic workup and therapy at an organ-specific level. Methods: Clinical features, laboratory findings, and response to treatment were reviewed retrospectively in an international cohort of 173 carriers of CTLA4 mutation. Patients were followed between 2014 and 2020 for a total of 2624 months from diagnosis. Clinical manifestations were grouped on the basis of organ-specific involvement. Medication use and response were recorded and evaluated. Results: Among the 173 CTLA4 mutation carriers, 123 (71%) had been treated for immune complications. Abatacept, rituximab, sirolimus, and corticosteroids ameliorated disease severity, especially in cases of cytopenias and lymphocytic organ infiltration of the gut, lungs, and central nervous system. Immunoglobulin replacement was effective in prevention of infection. Only 4 of 16 patients (25%) with cytopenia who underwent splenectomy had a sustained clinical response. Cure was achieved with stem cell transplantation in 13 of 18 patients (72%). As a result of the aforementioned methods, organ-specific treatment pathways were developed. Conclusion: Systemic immunosuppressants and abatacept may provide partial control but require ongoing administration. Allogeneic hematopoietic stem cell transplantation offers a possible cure for patients with CTLA-4 insufficiency.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Gastroenterologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Gastroenterology and Hepatology (hsv//eng)
Keyword
- CTLA-4; common variable immunodeficiency; primary immunodeficiency; diagnosis; treatment; abatacept; sirolimus; LRBA; rituximab; HSCT
Publication and Content Type
- ref (subject category)
- art (subject category)
Find in a library
To the university's database
- By the author/editor
-
Egg, David
-
Rump, Ina Caroli ...
-
Mitsuiki, Noriko
-
Rojas-Restrepo, ...
-
Maccari, Maria-E ...
-
Schwab, Charlott ...
-
show more...
-
Gabrysch, Annema ...
-
Warnatz, Klaus
-
Goldacker, Sigun ...
-
Patino, Virginia
-
Wolff, Daniel
-
Okada, Satoshi
-
Hayakawa, Seiich ...
-
Shikama, Yoshiak ...
-
Kanda, Kenji
-
Imai, Kohsuke
-
Sotomatsu, Manab ...
-
Kuwashima, Makot ...
-
Kamiya, Takahiro
-
Morio, Tomohiro
-
Matsumoto, Kazua ...
-
Mori, Takeshi
-
Yoshimoto, Yuri
-
Dybedal, Ingunn
-
Kanariou, Maria
-
Kucuk, Zeynep Ye ...
-
Chapdelaine, Hug ...
-
Petruzelkova, Le ...
-
Lorenz, Hanns-Ma ...
-
Sullivan, Kathle ...
-
Heimall, Jennife ...
-
Moutschen, Miche ...
-
Litzman, Jiri
-
Recher, Mike
-
Albert, Michael ...
-
Hauck, Fabian
-
Seneviratne, Sur ...
-
Schmid, Jana Pac ...
-
Kolios, Antonios
-
Unglik, Gary
-
Klemann, Christi ...
-
Snapper, Scott
-
Giulino-Roth, Li ...
-
Svaton, Michael
-
Platt, Craig D.
-
Hambleton, Sophi ...
-
Neth, Olaf
-
Gosse, Geraldine
-
Reinsch, Steffen
-
Holzinger, Dirk
-
Kim, Yae-Jean
-
Bakhtiar, Shahrz ...
-
Atschekzei, Fara ...
-
Schmidt, Reinhol ...
-
Sogkas, Georgios
-
Chandrakasan, Sh ...
-
Rae, William
-
Derfalvi, Beata
-
Marquart, Hanne ...
-
Ozen, Ahmet
-
Kiykim, Ayca
-
Karakoc-Aydiner, ...
-
Kralickova, Pavl ...
-
de Bree, Godelie ...
-
Kiritsi, Dimitra
-
Seidel, Markus G ...
-
Kobbe, Robin
-
Dantzer, Jennife ...
-
Alsina, Laia
-
Armangue, Thais
-
Lougaris, Vassil ...
-
Agyeman, Philipp
-
Nystrom, Sofia
-
Buchbinder, Davi ...
-
Arkwright, Peter ...
-
Grimbacher, Bodo
-
show less...
- About the subject
-
- MEDICAL AND HEALTH SCIENCES
-
MEDICAL AND HEAL ...
-
and Clinical Medicin ...
-
and Gastroenterology ...
- Articles in the publication
-
Journal of Aller ...
- By the university
-
Linköping University