{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,17]],"date-time":"2026-09-17T16:37:42Z","timestamp":1789663062097,"version":"4.0.1"},"reference-count":80,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2022,8,16]],"date-time":"2022-08-16T00:00:00Z","timestamp":1660608000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Immunol."],"abstract":"<jats:p>Primary Immunodeficiencies (PID) are a group of rare congenital disorders of the immune system. Autoimmune cytopenia (AIC) represents the most common autoimmune manifestation in PID patients. Treatment of AIC in PID patients can be really challenging, since they are often chronic, relapsing and refractory to first line therapies, thus requiring a broad variety of alternative therapeutic options. Moreover, immunosuppression should be fine balanced considering the increased susceptibility to infections in these patients. Specific therapeutic guidelines for AIC in PID patients are lacking. Treatment choice should be guided by the underlying disease. The study of the pathogenic mechanisms involved in the genesis of AIC in PID and our growing ability to define the molecular underpinnings of immune dysregulation has paved the way for the development of novel targeted treatments. Ideally, targeted therapy is directed against an overexpressed or overactive gene product or substitutes a defective protein, restoring the impaired pathway. Actually, the molecular diagnosis or a specific drug is not always available. However, defining the category of PID or the immunological phenotype can help to choose a semi-targeted therapy directed towards the suspected pathogenic mechanism. In this review we overview all the therapeutic interventions available for AIC in PID patients, according to different immunologic targets. In particular, we focus on T and\/or B cells targeting therapies. To support decision making in the future, prospective studies to define treatment response and predicting\/stratifying biomarkers for patients with AIC and PID are needed.<\/jats:p>","DOI":"10.3389\/fimmu.2022.911385","type":"journal-article","created":{"date-parts":[[2022,8,16]],"date-time":"2022-08-16T14:34:31Z","timestamp":1660660471000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":15,"title":["Targeted treatment of autoimmune cytopenias in primary immunodeficiencies"],"prefix":"10.3389","volume":"13","author":[{"given":"Lucia","family":"Pacillo","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuliana","family":"Giardino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donato","family":"Amodio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmela","family":"Giancotta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beatrice","family":"Rivalta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gioacchino Andrea","family":"Rotulo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma Concetta","family":"Manno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristina","family":"Cifaldi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Palumbo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudio","family":"Pignata","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo","family":"Palma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo","family":"Rossi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Finocchi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caterina","family":"Cancrini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2022,8,16]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"24","DOI":"10.1007\/s10875-019-00737-x","article-title":"Human inborn errors of immunity: 2019 update on the classification from the international union of immunological societies expert committee","volume":"40","author":"Tangye","year":"2020","journal-title":"J Clin Immunol"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2016.12.978","article-title":"Autoimmune and inflammatory manifestations occur frequently in patients with primary immunodeficiencies","volume":"140","author":"Fischer","year":"2017","journal-title":"J All Clin Immunol"},{"key":"B3","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1182\/blood-2018-11-887141","article-title":"Pediatric Evans syndrome is associated with a high frequency of potentially damaging variants in immune genes","volume":"134","author":"Hadjadj","year":"2019","journal-title":"Blood"},{"key":"B4","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2016.07.040","article-title":"P-CID study of the inborn errors working party of the ebmT. a prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis","volume":"139","author":"Speckmann","year":"2017","journal-title":"J Allergy Clin Immunol"},{"key":"B5","doi-asserted-by":"publisher","DOI":"10.1038\/nri2336","article-title":"Unravelling the association of partial T-cell immunodeficiency and immune dysregulation","volume":"8","author":"Liston","year":"2008","journal-title":"Nat Rev Immunol"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2019.02.038","article-title":"Outcomes and treatment strategies for autoimmunity and hyperinflammation in patients with RAG deficiency","volume":"7","author":"Farmer","year":"2019","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019000923","article-title":"Immune dysregulation in patients with RAG deficiency and other forms of combined immune deficiency","volume":"135","author":"Del Monte","year":"2020","journal-title":"Blood"},{"key":"B8","doi-asserted-by":"publisher","DOI":"10.1007\/s10875-021-01130-3","article-title":"Clinical, immunological, and molecular variability of rag deficiency: a retrospective analysis of 22 rag patients","volume":"42","author":"Cifaldi","year":"2021","journal-title":"J Clin Immunol"},{"key":"B9","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-03-259325","article-title":"How I treat autoimmune hemolytic anemias in adults","volume":"116","author":"Lechner","year":"2010","journal-title":"Blood"},{"key":"B10","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2010-08-302984","article-title":"The American society of hematology 2011 evidence-based practice guideline for immune thrombocytopenia","volume":"117","author":"Neunert","year":"2011","journal-title":"Blood"},{"key":"B11","doi-asserted-by":"publisher","DOI":"10.2450\/2016.0072-16","article-title":"Diagnosis and management of newly diagnosed childhood autoimmune haemolytic anaemia. recommendations from the red cell study group of the paediatric haemato-oncology Italian association","volume":"15","author":"Ladogana","year":"2017","journal-title":"Blood Transfus"},{"key":"B12","doi-asserted-by":"publisher","DOI":"10.2450\/2018.0024-18","article-title":"Second-line therapy in paediatric warm autoimmune haemolytic anaemia. guidelines from the associazione italiana onco-ematologia pediatrica (aieop)","volume":"16","author":"Ladogana","year":"2018","journal-title":"Blood Transfus"},{"key":"B13","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000966","article-title":"American Society of hematology 2019 guidelines of immune thrombocytopenia","volume":"3","author":"Neunert","year":"2019","journal-title":"Blood Adv"},{"key":"B14","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2019000812","article-title":"Updated international consensus report on the investigation and management of primary immune thrombocytopenia","volume":"3","author":"Provan","year":"2019","journal-title":"Blood Adv"},{"key":"B15","doi-asserted-by":"publisher","DOI":"10.1053\/j.seminhematol.2016.04.013","article-title":"Management of chronic immune thrombocytopenia in children and adolescents: lessons from an Austrian national cross-sectional study of 81 patients","volume":"53","author":"Sipurzynski","year":"2016","journal-title":"Semin Hematol"},{"key":"B16","year":""},{"key":"B17","doi-asserted-by":"publisher","DOI":"10.1126\/science.270.5234.286","article-title":"Immunosuppression by glucocorticoids: inhibition of nf-kb activity through inductionof ikb synthesis","volume":"270","author":"Auphan","year":"1995","journal-title":"Science"},{"key":"B18","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1007\/s00415-008-3002-0","article-title":"Advances in the understanding of the mechanism of action of IVIg","volume":"255","author":"Hartung","year":"2008","journal-title":"J Neurol"},{"key":"B19","doi-asserted-by":"publisher","DOI":"10.1016\/j.revmed.2012.05.007","article-title":"D\u00e9ficits immunitaires primitifs et cytop\u00e9nies auto-immunes de l\u2019adulte Primary immunodeficiencies presenting with autoimmune cytopenias in adults","volume":"34","author":"S\u00e8ve","year":"2013","journal-title":"Rev Med Interne"},{"key":"B20","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-06-583260","article-title":"Autoimmune and other cytopenias in primary immunodeficiencies: pathomechanisms, novel differential diagnoses, and treatment","volume":"15","author":"Seidel","year":"2014","journal-title":"Blood"},{"key":"B21","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2016.08.004","article-title":"Mechanism\u2019s based strategies for the management of autoimmunity and immunedysregulation in primary immunodeficiencies","volume":"4","author":"Walter","year":"2016","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B22","doi-asserted-by":"publisher","DOI":"10.1182\/hematology.2020000153","article-title":"Treatment of immune-mediated cytopenias in patients with primary immunodeficiencies and immune regulatory disorders (PIRDs)","author":"Seidel","year":"2020","journal-title":"Hematology"},{"key":"B23","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2018.12.017","article-title":"Mechanism-based precision therapy for the treatment of primary immunodeficiency and primary immunodysregulatory diseases. 2019 american academy of allergy, asthma & immunology","volume":"7","author":"Leiding","year":"2019","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B24","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2019.00353","article-title":"Inborn errors of immunity with immune dysregulation: from bench to bedside","volume":"7","author":"Delmonte","year":"2019","journal-title":"Front Pediatr"},{"key":"B25","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13533","article-title":"Mycophenolate mofetil and sirolimus as second or further line treatment in children with chronic refractory primitive or secondary autoimmune cytopenias: a single centre experience","volume":"171","author":"Miano","year":"2015","journal-title":"Br J Haematol"},{"key":"B26","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2005.05496.x","article-title":"Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome","volume":"129","author":"Rao","year":"2005","journal-title":"Br J Haematol"},{"key":"B27","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.11540","article-title":"Rituximab therapy for childhood Evans syndrome","volume":"92","author":"Bader-Meunier","year":"2007","journal-title":"Haematologica"},{"key":"B28","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2007.06616.x","article-title":"Rituximab for the treatment of autoimmune cytopenias in children with immune deficiency","volume":"138","author":"Kim","year":"2007","journal-title":"Br J Haematol"},{"key":"B29","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaip.2019.07.032","article-title":"Rituximab unveils hypogammaglobulinemia and immunodeficiency in children with autoimmune cytopenia","volume":"8","author":"Ottaviano","year":"2020","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B30","doi-asserted-by":"publisher","first-page":"498","DOI":"10.1111\/j.1365-2141.2011.08880.x","article-title":"Efficacy and safety of rituximab in common variable immunodeficiency-associated immune cytopenias: a retrospective multicentre study on 33 patients","volume":"155","author":"Gobert","year":"2011","journal-title":"Br J Haematol"},{"key":"B31","doi-asserted-by":"publisher","DOI":"10.1634\/theoncologist.8-6-508","article-title":"FDA Approval for the treatment of multiple myeloma progressing on prior therapy","volume":"8","author":"Kane","year":"2003","journal-title":"Oncologist"},{"key":"B32","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2016001412","article-title":"Bortezomib-based antibody depletion for refractory autoimmune hematological diseases","volume":"1","author":"Ratnasingam","year":"2016","journal-title":"Blood Adv"},{"key":"B33","doi-asserted-by":"publisher","first-page":"95","DOI":"10.1111\/imr.12389","article-title":"Monoclonal antibodies targeting CD38 in hematological malignancies and beyond","volume":"270","author":"van de Donk","year":"2016","journal-title":"Immunol Rev"},{"key":"B34","doi-asserted-by":"publisher","first-page":"1654","DOI":"10.1016\/j.bbmt.2014.06.032","article-title":"Bortezomib for refractory autoimmunity in pediatrics","volume":"20","author":"Khandelwal","year":"2014","journal-title":"Biol Blood Marrow Transplant"},{"key":"B35","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2014-206016","article-title":"The proteasome inhibitior bortezomib depletes plasma cells and ameliorates clinical manifestations of refractory systemic lupus erythematosus","volume":"74","author":"Alexander","year":"2015","journal-title":"Ann Rheum Dis"},{"key":"B36","doi-asserted-by":"publisher","first-page":"20406207211046428","DOI":"10.1177\/20406207211046428","article-title":"Bortezomib in autoimmune hemolytic anemia and beyond","volume":"12","author":"Pasquale","year":"2021","journal-title":"Ther Adv Hematol"},{"key":"B37","doi-asserted-by":"publisher","DOI":"10.1016\/j.hemonc.2019.05.006","article-title":"Bortezomib for immune thrombocytopenia and autoimmune hemolytic anemia","volume":"13","author":"Muhsen","year":"2020","journal-title":"Hematol Oncol Stem Cell Ther"},{"key":"B38","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1080\/09537104.2021.2002835","article-title":"Refractory immune thrombocytopenia successfully treated with bortezomib in a child with 22q11.2 deletion syndrome, complicated by Evans syndrome and hypogammaglobulinemia","volume":"8","author":"Conti","year":"2022","journal-title":"Platelets"},{"key":"B39","doi-asserted-by":"publisher","DOI":"10.1182\/bloodadvances.2018020883","article-title":"Daratumumab in life-threatening autoimmune hemolytic anemia following hematopoietic stem cell transplantation","volume":"2","author":"Schuetz","year":"2018","journal-title":"Blood Adv"},{"key":"B40","doi-asserted-by":"publisher","first-page":"4819","DOI":"10.1182\/blood.V128.22.4819.4819","article-title":"Daratumumab is effective in the treatment of refractory post-transplant autoimmune hemolytic anemia: a pediatric case report","volume":"128","author":"Tolbert","year":"2016","journal-title":"Blood"},{"key":"B41","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.17655","article-title":"Daratumumab in rituximab-refractory autoimmune haemolytic anaemia","volume":"194","author":"Rieger","year":"2021","journal-title":"Br J Haematol"},{"key":"B42","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2021.279232","article-title":"Daratumumab, an original approach for treating multi-refractory autoimmune cytopenia","volume":"106","author":"Crickx","year":"2021","journal-title":"Haematologica"},{"key":"B43","article-title":"The DART Study- Daratumumab Treatment in ITP","volume-title":"NCT04703621","year":"2021"},{"key":"B44","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(21)00592-4","article-title":"Isatuximab, carfilzomib, and dexamethasone in relapsed multiple myeloma (IKEMA): a multicentre, open-label, randomised phase 3 trial","volume":"397","author":"Moreau","year":"2021","journal-title":"Lancet"},{"key":"B45","year":"2020"},{"key":"B46","doi-asserted-by":"publisher","DOI":"10.3109\/03009742.2015.1074277","article-title":"Systemic lupus erythematosus exacerbation following cessation of belimumab treatment","volume":"45","author":"Furer","year":"2016","journal-title":"Scand J Rheumatol"},{"key":"B47","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2020.259481","article-title":"Efficacy, safety and immunological profile of combining rituximab with belimumab for adults with persistent or chronic immune thrombocytopenia: results from a prospective phase 2b trial","volume":"106","author":"Mah\u00e9vas","year":"2021","journal-title":"Haematologica"},{"key":"B48","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2013-205067","article-title":"Efficacy and safety of atacicept for prevention of flares in patients with moderate-to-severe systemic lupus erythematosus (SLE): 52-week data (APRIL-SLE randomised trial)","volume":"74","author":"Isenberg","year":"2015","journal-title":"Ann Rheum Dis"},{"key":"B49","doi-asserted-by":"publisher","DOI":"10.1038\/bcj.2017.5","article-title":"Ibrutinib in previously treated chronic lymphocytic leukemia patients with autoimmune cytopenias in the RESONATE study","volume":"7","author":"Montillo","year":"2017","journal-title":"Blood Cancer J"},{"key":"B50","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.15545","article-title":"Autoimmune cytopenias in patients with chronic lymphocytic leukaemia treated with ibrutinib in routine clinical practice at an academic medical centre","volume":"183","author":"Hampel","year":"2018","journal-title":"Br J Haematol"},{"key":"B51","doi-asserted-by":"publisher","DOI":"10.1002\/acr.22694","article-title":"Long-term safety and efficacy of epratuzumab in the treatment of moderate-to- severe systemic lupus erythematosus: results from an open-label extension study","volume":"68","author":"Wallace","year":"2016","journal-title":"Arthrit Care Res"},{"key":"B52","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2019.11.043","article-title":"Treatment with rapamycin can restore regulatory T-cell function in IPEX patients","volume":"145","author":"Passerini","year":"2020","journal-title":"J Allergy Clin Immunol"},{"key":"B53","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1182\/blood-2015-07-657981","article-title":"Sirolimus is effective in relapsed\/refractory autoimmune cytopenias: results of a prospective multi-institutional trial","volume":"127","author":"Bride","year":"2016","journal-title":"Blood"},{"key":"B54","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25119","article-title":"Sirolimus as a rescue therapy in children with immune thrombocytopenia refractory to mycophenolate mofetil","volume":"93","author":"Miano","year":"2018","journal-title":"Am J Hematol"},{"key":"B55","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2015-11-685024","article-title":"Hyperactive mTOR pathway promotes lymphoproliferation and abnormal differentiation in autoimmune lymphoproliferative syndrome","volume":"128","author":"V\u00f6lkl","year":"2016","journal-title":"Blood"},{"key":"B56","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2018.02.055","article-title":"Phenotype, penetrance, and treatment of 133 cytotoxic T-lymphocyte antigen 4-insufficient subjects","volume":"142","author":"Schwab","year":"2018","journal-title":"J Allergy Clin Immunol"},{"key":"B57","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1016\/j.jaci.2015.08.036","article-title":"Abatacept alleviates severe autoimmune symptoms in a patient carrying a de novo variant in CTLA-4","volume":"137","author":"Lee","year":"2016","journal-title":"J Allergy Clin Immunol"},{"key":"B58","doi-asserted-by":"publisher","first-page":"2790","DOI":"10.1016\/j.jaip.2019.06.011","article-title":"Abatacept as a long-term targeted therapy for LRBA deficiency","volume":"7","author":"Kiykim","year":"2019","journal-title":"J Allergy Clin Immunol Pract"},{"key":"B59","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2017-08-801191","article-title":"Effective \u201cactivated PI3K\u03b4 syndrome\u201d-targeted therapy with the PI3K\u03b4 inhibitor leniolisib","volume":"130","author":"Rao","year":"2017","journal-title":"Blood"},{"key":"B60","doi-asserted-by":"publisher","DOI":"10.4049\/jimmunol.2000326","article-title":"Seletalisib for activated PI3K\u03b4 syndromes: open-label phase 1b and extension studies","volume":"205","author":"Diaz","year":"2020","journal-title":"J Immunol"},{"key":"B61","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2018.07.020","article-title":"Jakinibs for the treatment of immune dysregulation in patients with gain-of-function signal transducer and activator of transcription 1 (STAT1) or STAT3 mutations","volume":"142","author":"Forbes","year":"2018","journal-title":"J Allergy Clin Immunol"},{"key":"B62","doi-asserted-by":"publisher","first-page":"1629","DOI":"10.1016\/j.jaci.2016.11.022","article-title":"Ruxolitinib reverses dysregulated T helper cell responses and controls autoimmunity caused by a novel signal transducer and activator of transcription 1 (STAT1) gain-of-function mutation","volume":"139","author":"Weinacht","year":"2017","journal-title":"J Allergy Clin Immunol"},{"key":"B63","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1016\/j.coi.2016.10.001","article-title":"Targeting cytokine signaling in autoimmunity: back to the future and beyond","volume":"43","author":"Hirahara","year":"2016","journal-title":"Curr Opin Immunol"},{"key":"B64","doi-asserted-by":"publisher","first-page":"784","DOI":"10.1016\/j.phrs.2016.07.038","article-title":"Janus kinase (JAK) inhibitors in the treatment of inflammatory and neoplastic diseases","volume":"111","author":"Roskoski","year":"2016","journal-title":"Pharmacol Res"},{"key":"B65","doi-asserted-by":"publisher","first-page":"622","DOI":"10.1016\/j.jaci.2016.03.007","article-title":"A novel genomic inversion in Wiskott-Aldrich\u2013associated autoinflammation","volume":"138","author":"Brigida","year":"2016","journal-title":"J Allergy Clin Immunol"},{"key":"B66","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-4642(20)30304-7","article-title":"A national consensus management pathway for paediatric inflammatory multisystem syndrome temporally associated with COVID-19 (PIMS-TS): results of a national Delphi process","volume":"5","author":"Harwood","year":"2021","journal-title":"Lancet Child Adolesc Health"},{"key":"B67","doi-asserted-by":"publisher","DOI":"10.1016\/S2352-4642(20)30177-2","article-title":"COVID-19 in children and adolescents in Europe: a multinational, multicentre cohort study","volume":"4","author":"G\u00f6tzinger","year":"2020","journal-title":"Lancet Child Adolesc Health"},{"key":"B68","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/keaa837","article-title":"Anti-tumour necrosis factor treatment for the prevention of ischaemic events in patients with deficiency of adenosine deaminase 2 (DADA2)","volume":"60","author":"Cooray","year":"2021","journal-title":"Rheumatol (Oxford)"},{"key":"B69","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1808575","article-title":"Plerixafor for the treatment of WHIM syndrome","volume":"380","author":"McDermott","year":"2019","journal-title":"N Engl J Med"},{"key":"B70","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2013-09-527226","article-title":"A phase 1 clinical trial of long-term, low-dose treatment of WHIM syndrome with the CXCR4 antagonist plerixafor","volume":"123","author":"McDermott","year":"2014","journal-title":"Blood"},{"key":"B71","doi-asserted-by":"publisher","first-page":"2994","DOI":"10.1182\/blood.2020007197","article-title":"Results of a phase 2 trial of an oral CXCR4 antagonist, mavorixafor, for treatment of WHIM syndrome","volume":"136","author":"Dale","year":"2020","journal-title":"Blood"},{"key":"B72","doi-asserted-by":"publisher","first-page":"9181698","DOI":"10.1155\/2016\/9181698","article-title":"Refractory IgG warm autoimmune hemolytic anemia treated with eculizumab: a novel application of anticomplement therapy","volume":"2016","author":"Ma","year":"2016","journal-title":"Case Rep Hematol"},{"key":"B73","doi-asserted-by":"publisher","DOI":"10.1002\/ccr3.3250","article-title":"Efficacy of eculizumab in refractory life-threatening warm autoimmune hemolytic anemia associated with chronic myelomonocytic leukemia","volume":"8","author":"Gauchy","year":"2020","journal-title":"Clin Case Rep"},{"key":"B74","doi-asserted-by":"publisher","DOI":"10.1191\/0961203305lu2109oa","article-title":"Mechanisms of action of mycophenolate mofetil","author":"Allison","year":"2005","journal-title":"Lupus"},{"key":"B75","doi-asserted-by":"publisher","DOI":"10.1016\/j.jhep.2020.07.040","article-title":"Epidemiological pattern, incidence, and outcomes of COVID-19 in liver transplant patients","volume":"74","author":"Colmenero","year":"2021","journal-title":"J Hepatol"},{"key":"B76","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2017.12.1000","article-title":"Autonomous role of wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation","volume":"142","author":"Sereni","year":"2018","journal-title":"J Allergy Clin Immunol"},{"key":"B77","doi-asserted-by":"publisher","DOI":"10.1038\/s41409-021-01378-8","article-title":"Inborn errors working party of the european society for blood and marrow transplantation and the european society for immune deficiencies, and european reference network on rare primary immunodeficiency autoinflammatory autoimmune diseases (rita). ebmt\/esid inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity","volume":"56","author":"Lankester","year":"2021","journal-title":"Bone Marrow Transpl"},{"key":"B78","doi-asserted-by":"publisher","DOI":"10.1007\/s10875-021-01098-0","article-title":"Hematopoietic cell transplantation cures adenosine deaminase 2 deficiency: report on 30 patients","volume":"41","author":"Hashem","year":"2021","journal-title":"J Clin Immunol"},{"key":"B79","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2019.00443","article-title":"Autologous stem-cell-based gene therapy for inherited disorders: state of the art and perspectives","volume":"7","author":"Staal","year":"2019","journal-title":"Front Pediatr"},{"key":"B80","doi-asserted-by":"publisher","first-page":"71","DOI":"10.1038\/s41591-021-01641-x","article-title":"M. long-term safety and efficacy of lentiviral hematopoietic stem\/progenitor cell gene therapy for wiskott-Aldrich syndrome","volume":"28","author":"Magnani","year":"2022","journal-title":"Nat Med"}],"container-title":["Frontiers in Immunology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fimmu.2022.911385\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,8,16]],"date-time":"2022-08-16T14:34:35Z","timestamp":1660660475000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fimmu.2022.911385\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,8,16]]},"references-count":80,"alternative-id":["10.3389\/fimmu.2022.911385"],"URL":"https:\/\/doi.org\/10.3389\/fimmu.2022.911385","relation":{},"ISSN":["1664-3224"],"issn-type":[{"value":"1664-3224","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,8,16]]},"article-number":"911385"}}