Off-Label Use of Intravenous Immunoglobulin for Hematological Conditions: A Review of Clinical Effectiveness


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Health Technology Review
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Summary with Critical Appraisal
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  1. What is the clinical effectiveness of off-label use of intravenous or subcutaneous immunoglobulin for the treatment of hematological conditions?

Key Message

Four studies met the inclusion criteria for this report. One systematic review reported on patients with fetal and neonatal alloimmune thrombocytopenia (FNAIT) and one systematic review, one randomized controlled study (RCT) and one non-randomized study included patients with hemolytic disease of the fetus and neonate (HDFN). The studies compared intravenous immunoglobulin (IVIG) with or without corticosteroids to no treatment, placebo, or a combination of corticosteroids, phototherapy, exchange transfusion (ET), fetal blood sampling, and intrauterine platelet transfusion (IUPT). The results of this report must be interpreted with caution given the heterogeneity observed among the included studies, as well as some limitations in study quality.Overall, the evidence on the effectiveness of off-label use of IVIG for hematological conditions was mixed. Compared with FNAIT patients who were treated with corticosteroids or IUPT, a smaller proportion of patients on IVIG had low platelet counts (i.e.,