Evolution of disease activity and biomarkers on and off rapamycin in 28 patients with autoimmune lymphoproliferative syndrome

Christian Klemann; Myrian Esquivel; Aude Magerus-Chatinet; Myriam R Lorenz; Ilka Fuchs; Nathalie Neveux; Martin Castelle; Jan Rohr; Claudia Bettoni da Cunha; Martin Ebinger; Robin Kobbe; Bernhard Kremens; Florian Kollert; Eleonora Gambineri; Kai Lehmberg; Markus G Seidel; Kathrin Siepermann; Thomas Voelker; Volker Schuster; Sigune Goldacker; Klaus Schwarz; Carsten Speckmann; Capucine Picard; Alain Fischer; Frederic Rieux-Laucat; Stephan Ehl; Anne Rensing-Ehl; Benedicte Neven

doi:10.3324/haematol.2016.153411

Evolution of disease activity and biomarkers on and off rapamycin in 28 patients with autoimmune lymphoproliferative syndrome

Haematologica. 2017 Feb;102(2):e52-e56. doi: 10.3324/haematol.2016.153411. Epub 2016 Oct 27.

Authors

Christian Klemann^{1

2}, Myrian Esquivel^{3

4}, Aude Magerus-Chatinet^{3

5}, Myriam R Lorenz^{6

7}, Ilka Fuchs¹, Nathalie Neveux⁸, Martin Castelle⁴, Jan Rohr^{1

2}, Claudia Bettoni da Cunha⁹, Martin Ebinger¹⁰, Robin Kobbe¹¹, Bernhard Kremens¹², Florian Kollert¹³, Eleonora Gambineri¹⁴, Kai Lehmberg¹⁵, Markus G Seidel¹⁶, Kathrin Siepermann¹⁷, Thomas Voelker¹⁸, Volker Schuster¹⁹, Sigune Goldacker¹, Klaus Schwarz^{6

7}, Carsten Speckmann^{1

2}, Capucine Picard^{3

4

20}, Alain Fischer^{3

4

21}, Frederic Rieux-Laucat^{3

5}, Stephan Ehl^{1

2}, Anne Rensing-Ehl²², Benedicte Neven²³

Affiliations

¹ Center for Chronic Immunodeficiency (CCI), Medical Center, Faculty of Medicine, University of Freiburg, Germany.
² Center for Pediatrics, Department of Pediatric Hematology and Oncology, University Medical Center, Faculty of Medicine, University of Freiburg, Germany.
³ INSERM UMR 1163, Laboratory of the immunogenetics of Pediatric Autoimmune Diseases, Paris, France.
⁴ Unite d'Immuno Hematologie Pediatrique, Hopital Necker-Enfants Malades, Assistance Publique des Hopitaux de Paris, France.
⁵ Paris Descartes - Sorbonne Paris Cité University, Imagine Institute, France.
⁶ Institute for Transfusion Medicine, University of Ulm, Germany.
⁷ Institute for Clinical Transfusion Medicine and Immunogenetics Ulm, German Red Cross Blood Service, Germany.
⁸ Service de Biochimie, Hopital Cochin, Assistance Publique Hopitaux de Paris, France.
⁹ Department of Pediatric Hematology and Oncology, Hannover Medical School, Germany.
¹⁰ University Children's Hospital Tübingen, Germany.
¹¹ Department of Pediatrics, Infectious Diseases and Immunity, University Medical Centre Hamburg-Eppendorf, Germany.
¹² Department of Rheumatology and Clinical Immunology, University Medical Centre Freiburg, Germany.
¹³ University Hospital Essen, Department of Paediatrics III, University of Duisburg-Essen, Germany.
¹⁴ Department of "NEUROFARBA": Section of Child's Health, University of Florence, Department of Haematology-Oncology: BMT Unit, Department of Fetal and Neonatal Medicine: Rare Diseases, "Anna Meyer" Children's Hospital, Florence, Italy.
¹⁵ Department of Pediatric Haematology/Oncology, University Medical Centre Hamburg, Germany.
¹⁶ Division of Pediatric Hematology-Oncology, Department of Pediatrics and Adolescent Medicine, Medical University Graz, Austria.
¹⁷ Department of Pediatric and Adolescent Medicine, HELIOS Klinikum Krefeld, Germany.
¹⁸ Hospital for Pediatric Haematology and Oncology, Medical Center Kassel GmbH, Germany.
¹⁹ Hospital for Children and Adolescents, University of Leipzig, Germany.
²⁰ Centre d'Etude des Deficits Immunitaires, Hopital Necker-Enfants Malades, Assistance Publique des Hopitaux de Paris, France.
²¹ College de France, Paris, France.
²² Center for Chronic Immunodeficiency (CCI), Medical Center, Faculty of Medicine, University of Freiburg, Germany benedicte.neven@aphp.fr anne.rensing-ehl@uniklinik-freiburg.de.
²³ Unite d'Immuno Hematologie Pediatrique, Hopital Necker-Enfants Malades, Assistance Publique des Hopitaux de Paris, France benedicte.neven@aphp.fr anne.rensing-ehl@uniklinik-freiburg.de.

No abstract available

Publication types

Letter
Multicenter Study
Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Antibiotics, Antineoplastic / therapeutic use*
Autoimmune Lymphoproliferative Syndrome / drug therapy*
Autoimmune Lymphoproliferative Syndrome / genetics
Autoimmune Lymphoproliferative Syndrome / immunology
Autoimmune Lymphoproliferative Syndrome / pathology
Biomarkers / metabolism
Child
Child, Preschool
Disease Progression
Drug Administration Schedule
Fas Ligand Protein / genetics
Fas Ligand Protein / immunology
Female
Gene Expression Regulation
Humans
Immunosuppressive Agents / therapeutic use*
Infant
Infant, Newborn
Interleukin-10 / genetics
Interleukin-10 / immunology
Lymphadenopathy / drug therapy
Lymphadenopathy / genetics
Lymphadenopathy / immunology
Lymphadenopathy / pathology
Male
Retrospective Studies
Sirolimus / therapeutic use*
Splenomegaly / drug therapy
Splenomegaly / genetics
Splenomegaly / immunology
Splenomegaly / pathology
fas Receptor / genetics*
fas Receptor / immunology

Substances

Antibiotics, Antineoplastic
Biomarkers
FAS protein, human
FASLG protein, human
Fas Ligand Protein
IL10 protein, human
Immunosuppressive Agents
fas Receptor
Interleukin-10
Sirolimus