| خلاصه مقاله | Chronic immune thrombocytopenia (ITP) is a significant challenge in the field of pediatric hematology. Among children diagnosed with acute ITP, approximately 10-15% of patients continue to experience a decrease in platelet count and various forms of bleeding after 6-12 months, despite first and second-line therapies. The approach and choice of medications in the management of chronic ITP in children, alongside splenectomy and Rituximab use, have been topics of discussion in international guidelines. Recently, the use of Romiplostim before proceeding with splenectomy or initiating Rituximab has been included in many treatment guidelines for chronic ITP in children. In the latest guideline published by the American Society of Hematology (ASH), Romiplostim, a Thrombopoietin receptor agonist, has been accepted as a second-line therapeutic option for chronic ITP in children.
This study aims to investigate the impact and role of age in the therapeutic response to Romiplostim in children with chronic ITP. The significance of age as a general parameter in distinguishing between childhood and adulthood has been widely recognized through numerous clinical trials in adults and children. In our experience, over several years of using Romiplostim, we have observed that age plays a crucial role in the treatment response of children to this medication. This cross-sectional study was conducted between March 2018 and April 2022 at the Children’s Hospital in Tabriz to shed light on the role of age in the therapeutic response to Romiplostim. Forty-seven patients, with an average age of 8.11 years (1.5-17.9), who had been diagnosed with ITP for at least 6 months and had a platelet count of less than 30,000/mm3, were enrolled in the study. None of the study subjects responded adequately to first and second-line medications. Additionally, splenectomy or Rituximab administration was not performed in any of the study participants either due to their age or parental refusal.
The patients had a history of repeated treatment with corticosteroids, IVIG, Intravenous anti-D immunoglobulin, and cyclosporine, but their response to treatment was poor or refractory. In our study, Romiplostim was initiated at a subcutaneous dose of 1 mcg/kg weekly for all patients. The patients were divided into three age groups: less than 5 years, 5 to 10 years, and 10 to 18 years. The primary objective of treatment was to achieve a weekly platelet count ≥50,000 mm3 and maintain this increase for the next 24 weeks. A positive initial treatment response, with platelet counts reaching above 50,000/mm3, was observed in 37 patients. However, in ten patients, despite increasing the weekly dose of Romiplostim for four consecutive weeks, no increase in platelet count was observed. Furthermore, 26 patients exhibited a sustained increase in platelet count above 50,000/mm3 in the subsequent 24 weeks. Notably, the initial treatment response and sustained increase in platelet count were significantly higher in the age group over ten years compared to children under five years. Moreover, the age group of 5 to 10 years demonstrated a significantly better sustained increase in platelet count compared to the age group under five years. Throughout the study, no life-threatening bleeding or thrombotic events were reported in any age group. Furthermore, none of the study participants showed a platelet count exceeding 400,000/mm3. The most common adverse drug reaction observed in this study was fever, upper respiratory tract infections, and skin rash, with no significant differences among the age groups.
In conclusion, according to the authors of this article, Romiplostim demonstrated a sustained treatment response in at least 50 percent of the study patients, with a more significant therapeutic response observed in children over ten years of age. |