Use of Thrombopoietin-Receptor Agonist in CVID-Associated Immune Thrombocytopenia

Carrabba, Maria; Barcellini, Wilma; Gavarini, Fabio

doi:10.1007/s10875-016-0282-5

Cited by 15 publications

(11 citation statements)

References 11 publications

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“…19 Newer options for cytopenias include thrombopoietin-receptor agonists, which have also shown benefit. 20 In many cases, ITP or AIHA is accompanied by an enlarged spleen or cervical, mediastinal, or abdominal lymphadenopathy. However, splenectomy in patients with CVID should be avoided given the susceptibility to postoperative infections and sepsis with encapsulated organisms.…”

Section: Autoimmune Hematologic Diseasementioning

confidence: 99%

Autoimmunity in common variable immunodeficiency

Agarwal

Cunningham‐Rundles

2019

Annals of Allergy, Asthma & Immunology

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Key MessagesBesides infections, autoimmune or inflammatory diseases are common in patients with primary immunodeficiency. Common organspecific autoimmunity in CVID patients include the gastrointestinal tract, lung, hematological system, joints, and skin.The onset of autoimmune disease in CVID varies, as does the severity and progression.Treatment of autoimmunity is often complicated by drug adverse effects and prolonged immunosuppression but includes high-dose immunoglobulin, corticosteroids, selected immunosuppressants, and other immune modulators.The mechanisms of these autoimmune/inflammatory diseases are still being investigated but likely involve defects in central and peripheral tolerance and autoreactive T and B cells. A B S T R A C TObjective: Common variable immunodeficiency (CVID) is a primary immunodeficiency that is clinically heterogeneous, characterized by both infectious and noninfectious complications. Although the hallmark of disease presentation is commonly a history of recurrent sinopulmonary infections, autoimmunity and noninfectious inflammatory conditions are increasingly associated with CVID. Data Sources: A comprehensive literature search using PubMed of basic science and clinical articles was performed. Study Selections: Articles discussing the association of autoimmunity with primary immunodeficiency, specifically CVID, were selected. Results: The most common autoimmune conditions are cytopenias, including immune thrombocytopenia purpura and hemolytic anemia, but organ-specific autoimmune/inflammatory complications involving the gastrointestinal, skin, joints, connective tissue, and respiratory tract. In most cases, immunoglobulin replacement therapy does not ameliorate or treat these inflammatory complications, and additional immunomodulatory treatments are needed. Conclusion: Mechanisms producing these conditions are poorly understood but include cytokine and cellular inflammatory pathways, and loss of tolerance to self-antigens through the multiple signaling molecules and pathways common to tolerance and immune deficiency.

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Section: Autoimmune Hematologic Diseasementioning

confidence: 99%

Autoimmunity in common variable immunodeficiency

Agarwal

Cunningham‐Rundles

2019

Annals of Allergy, Asthma & Immunology

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“…In one retrospective multicenter study, rituximab was highly effective 33 patients with CVID‐associated immune cytopenias, leading to an initial response rate of 80 % and a sustained response rate of 50%, after a mean follow up of 39 ± 30 months following the first administration. Other more recent options for cytopenias include thrombopoietin‐receptor agonists, which have also shown benefit . However, in some cases splenectomy has already been performed, usually for treatment of cytopenias, but also for an increasingly large spleen and/or concern for lymphoma.…”

Section: Treatmentmentioning

confidence: 99%

“…Other more recent options for cytopenias include thrombopoietin-receptor agonists, which have also shown benefit. 170 However, in some cases splenectomy has already been performed, usually for treatment of cytopenias, but also for an increasingly large spleen and/or concern for lymphoma. While rituximab has largely supplanted splenectomy for resolution of cytopenias in many patients, it has often been performed before the diagnosis of CVID.…”

Section: Tre Atmentmentioning

confidence: 99%

Common variable immune deficiency: Dissection of the variable

Cunningham‐Rundles

2018

Immunological Reviews

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Summary Starting about 60 years ago, a number of reports appeared that outlined the severe clinical course of a few adult subjects with profound hypogammaglobinemia. Puzzled by the lack of family history and adult onset of symptoms in most, the name “acquired” hypogammaglobinemia was given, but later altered to the current name common variable immune deficiency (CVID). Pathology reports remarked on the loss of lymph node architecture and paucity of plasma cells in lymphoid tissues in these subjects. While characterized by reduced serum IgG and IgA and often IgM, and thus classified amongst the B cell defects, an increasing number of cellular defects in these patients have been recognized over time. In the early years, severe respiratory tract infections commonly led to a shortened life span, but the wide spread availability of immune globulin concentrates for the last 25 years has improved survival. However, chronic non-infectious inflammatory and autoimmune conditions have now emerged as challenging clinical problems; these require further immunologic understanding and additional therapeutic measures. Recent study of this phenotypic syndrome have provided an increasingly fertile ground for the identification of autosomal recessive and now more commonly, autosomal dominant gene defects which lead to the loss of B cell development in this syndrome.

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“…To date, abatacept appears to be the most promising therapy for this condition and some patients have been successfully treated for 5–8 years . The increased use of thrombopoietin mimetics romiplostim and eltrombopag for immune‐mediated thrombocytopenia offer another option for patients manifesting with thrombocytopenia . Additionally, HSCT may be a consideration in carefully selected patients …”

Section: Common Variable Immunodeficiencymentioning

confidence: 99%

“…59 The increased use of thrombopoietin mimetics romiplostim and eltrombopag for immunemediated thrombocytopenia 62,63 offer another option for patients manifesting with thrombocytopenia. 64 Additionally, HSCT may be a consideration in carefully selected patients. 65 We recently evaluated a 13-year-old male with history of Evans syndrome, autoimmune colitis, lymphadenitis, and uveitis who presented for a second opinion of refractory left-sided neck pain and swelling.…”

Section: Lrba Deficiencymentioning

confidence: 99%

Diagnosis and management of chronic and refractory immune cytopenias in children, adolescents, and young adults

Rotz

Ware

Kumar

2018

Pediatric Blood & Cancer

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Children, adolescents, and young adults with chronic refractory autoimmune cytopenias represent a rare but challenging group of patients, who are managed frequently by pediatric hematologists. Novel diagnostic tests and genomic discoveries are refining historical diagnoses of Evans syndrome and common variable immunodeficiency, while also elucidating the cellular and molecular basis for these disorders. Genetic characterization of chronic and refractory autoimmune cytopenias has led to targeted therapies with improved clinical outcomes and fewer off-target toxicities. In this review, we focus on the appropriate diagnostic workup, expanded genetic testing, and novel treatment opportunities that are available for these challenging patients.

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Use of Thrombopoietin-Receptor Agonist in CVID-Associated Immune Thrombocytopenia

Cited by 15 publications

References 11 publications

Autoimmunity in common variable immunodeficiency

Autoimmunity in common variable immunodeficiency

Common variable immune deficiency: Dissection of the variable

Diagnosis and management of chronic and refractory immune cytopenias in children, adolescents, and young adults

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