Pediatric autoimmune hemolytic anemia: A single-center retrospective study.

BACKGROUND: Autoimmune hemolytic anemia (AIHA) is a rare, life-threatening disease in pediatrics. This article describes the clinical features, diagnostic workup, treatment and outcome in patients with AIHA. METHOD: Medical charts of under 18-year-old patients with AIHA treated at a tertiary Brazilian institution from 2006 to 2021 were retrospectively reviewed. Data analysis was primarily descriptive, using medians, interquartile ranges, and categorical variables presented as absolute frequencies. MAIN RESULTS: Twenty-four patients (14 female, 10 male) were evaluated in this study. The median age at diagnosis was 5.99 years (range: 0.25-17.1 years) and the median hemoglobin level was 4.85 g/dL (range: 4.17-5.57 g/dL). Most had warm antibodies (83.3 %). Twelve patients (50 %) had known underlining diseases, four (16.6 %) presented with AIHA concomitant with acute infectious diseases and three (12.5 %) had an undetermined post-vaccine association. Steroids and intravenous immunoglobulin were first-line therapy in 23 cases. Seven patients (29.1 %) required second and third-line treatments (rituximab, cyclophosphamide and splenectomy). The median follow-up period was 4.4 years (range: 1.0-6.7 years). Thirteen patients (54.1 %) were discharged, five cases (20.8 %) were lost to follow-up and no patient died. The median age for the six remaining patients was 11.53 years (8.5-14.7) with all of them having complete responses with no further therapies. CONCLUSION: Most cases of AIHA are secondary to an underlying systemic disease or have a possible correlation with infections/vaccines and respond to steroids. The second and third-line therapies for refractory and relapse cases remain a dilemma. A prospective, multicenter study is essential to address the best therapeutic combinations.

Safety and efficacy of romiplostim in children and adolescents with Immune thrombocytopenia: A systematic review.

OBJECTIVE: To evaluate the efficacy and safety of romiplostim (thrombopoietin-receptor agonist) in the treatment of pediatric immune thrombocytopenia (ITP). METHODS: Searches were conducted in MEDLINE, EMBASE, LILACS, Cochrane Central Register of Controlled Trials and ClinicalTrials.gov (from January 2011 to August 2021). Randomized controlled trials (RCTs), double-blind, comparing romiplostim with a placebo in pediatric persistent or chronic ITP were included. The primary outcome was the overall response rate (platelets ≥ 50 × 109/L) in the absence of rescue therapy for at least two consecutive weeks. The secondary endpoints were the minimization of clinically significant bleeding and the necessity for rescue treatments and the maximization of safety (incidence of overall adverse events) and durable response (maintaining platelet counts for at least twelve weeks). RESULTS: Two double-blind randomized placebo-controlled trials (84 participants) were included in this systematic review. Our data showed that, compared to the placebo group, the proportion of patients achieving durable platelet response was significantly higher in the romiplostim group (p = 0.003, RR = 6.34, 95%CI = 1.89 - 21.23), as was the overall response in the romiplostim group (p = 0.002, RR = 3.62, 95%CI = 1.63 - 8.03). Significant bleeding incidents (p = 0.49), overall adverse events (p = 0.71) and the need for rescue treatment (p = 0.13) were not statistically different between the romiplostim and placebo groups. CONCLUSIONS: Romiplostim might improve both durable and overall platelet response in children and adolescents with ITP, compared to a placebo. More clinical trials are needed to evaluate the efficacy and safety of romiplostim and to compare it with other second-line treatments that are being used in pediatric ITP.

Safety and efficacy of romiplostim in children and adolescents with Immune thrombocytopenia: A systematic review

Abstract Objective To evaluate the efficacy and safety of romiplostim (thrombopoietin-receptor agonist) in the treatment of pediatric immune thrombocytopenia (ITP). Methods Searches were conducted in MEDLINE, EMBASE, LILACS, Cochrane Central Register of Controlled Trials and ClinicalTrials.gov (from January 2011 to August 2021). Randomized controlled trials (RCTs), double-blind, comparing romiplostim with a placebo in pediatric persistent or chronic ITP were included. The primary outcome was the overall response rate (platelets ≥ 50 × 109/L) in the absence of rescue therapy for at least two consecutive weeks. The secondary endpoints were the minimization of clinically significant bleeding and the necessity for rescue treatments and the maximization of safety (incidence of overall adverse events) and durable response (maintaining platelet counts for at least twelve weeks). Results Two double-blind randomized placebo-controlled trials (84 participants) were included in this systematic review. Our data showed that, compared to the placebo group, the proportion of patients achieving durable platelet response was significantly higher in the romiplostim group (p= 0.003, RR = 6.34, 95%CI = 1.89 - 21.23), as was the overall response in the romiplostim group (p= 0.002, RR = 3.62, 95%CI = 1.63 - 8.03). Significant bleeding incidents (p= 0.49), overall adverse events (p= 0.71) and the need for rescue treatment (p= 0.13) were not statistically different between the romiplostim and placebo groups. Conclusions Romiplostim might improve both durable and overall platelet response in children and adolescents with ITP, compared to a placebo. More clinical trials are needed to evaluate the efficacy and safety of romiplostim and to compare it with other second-line treatments that are being used in pediatric ITP.