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Diagnosis and management of autoimmune cytopenias in childhood.

ABSTRACT: The diagnosis and management of children with autoimmune cytopenias can be challenging. Children can present with immune-mediated destruction of a single-cell lineage or multiple cell lineages, including platelets (immune thrombocytopenia [ITP]), erythrocytes (autoimmune hemolytic anemia), and neutrophils (autoimmune neutropenia). Immune-mediated destruction can be primary or secondary to a comorbid immunodeficiency, malignancy, rheumatologic condition, or lymphoproliferative disorder. Treatment options generally consist of nonspecific immune suppression or modulation. This nonspecific approach is changing as recent insights into disease biology have led to targeted therapies, including the use of thrombopoietin mimetics in ITP and sirolimus for cytopenias associated with autoimmune lymphoproliferative syndrome.

SUBMITTER: Teachey DT

PROVIDER: S-EPMC5384653 | biostudies-literature | 2013 Dec

REPOSITORIES: biostudies-literature

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Diagnosis and management of autoimmune cytopenias in childhood.

Teachey David T DT Lambert Michele P MP

Pediatric clinics of North America 20131005 6

The diagnosis and management of children with autoimmune cytopenias can be challenging. Children can present with immune-mediated destruction of a single-cell lineage or multiple cell lineages, including platelets (immune thrombocytopenia [ITP]), erythrocytes (autoimmune hemolytic anemia), and neutrophils (autoimmune neutropenia). Immune-mediated destruction can be primary or secondary to a comorbid immunodeficiency, malignancy, rheumatologic condition, or lymphoproliferative disorder. Treatment ...[more]

PMID: 24237984

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Project description:Background: Autoimmune cytopenias (AICs) are potentially life-threatening complications following hematopoietic cell transplantation (HCT), yet little is understood about the mechanism by which they develop. We hypothesized that discordant B cell and T cell recovery is associated with AICs in transplant patients, and that this might differ based on transplant indication. Methods: In this case control study of children who underwent HCT at our institution, we evaluated the clinical and transplant characteristics of subjects who developed AICs compared to a control group matched by transplant indication and donor type. In cases, we analyzed the state of immune reconstitution, including B cell recovery, T cell recovery, and chimerism, immediately prior to AIC onset. Subjects were stratified by primary indication for transplant as malignancy (n = 7), primary immune deficiency (PID, n = 9) or other non-malignant disease (n = 4). We then described the treatment and outcomes for 20 subjects who developed AICs. Results: In our cohort, cases were older than controls, were more likely to receive a myeloablative conditioning regimen and had a significantly lower prevalence of chronic GVHD. There were distinct differences in the state of immune recovery based on transplant indication. None of the patients (0/7) transplanted for primary malignancy had T cell recovery at AIC onset compared to 71% (5/7) of patients with PID and 33% (1/3) of patients with non-malignant disease. The subset of patients with PID and non-malignant disease who achieved T cell reconstitution (6/6) prior to AIC onset, all demonstrated mixed or split chimerism. Subjects with AIHA or multi-lineage cytopenias had particularly refractory courses with poor treatment response to IVIG, steroids, and rituximab. Conclusions: These results highlight the heterogeneity of AICs in this population and suggest that multiple mechanisms may contribute to the development of post-transplant AICs. Patients with full donor chimerism may have early B cell recovery without proper T cell regulation, while patients with mixed or split donor chimerism may have residual host B or plasma cells making antibodies against donor blood cells. A prospective, multi-center trial is needed to develop personalized treatment approaches that target the immune dysregulation present and improve outcomes in patients with post-transplant AICs.

Dataset Information

Diagnosis and management of autoimmune cytopenias in childhood.

Publications

Diagnosis and management of autoimmune cytopenias in childhood.

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