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Background: There is limited information on the natural course of chronic ITP in children. We aimed to evaluate the clinical and demographic characteristics of children with chronic ITP in the era before the availability of eltrombopag. Methods: A total of 86 children with chronic ITP between 1978–2014 were included. Demographic findings, laboratory results, clinical signs, bleeding scores, response time and time of complete remission were recorded. Results: The male/female ratio was 1.09, and median follow-up time was 3 years (range: 1.5–17 years). The median age at diagnosis of chronic ITP was 7 years (range: 2–17), and the median initial platelet count was 10 × 109/L (range: 1–66 × 109/L). Petechiae/ecchymoses were the most common clinical sign (86%) and followed by mucosal bleeding (39.5%). Severe bleeding was seen in 5% of the patients. None of them had intracranial hemorrhage. Twenty patients underwent splenectomy, and the rate of complete remission was 70%. Spontaneous complete remission was seen in 29% of the patients, and the median time to spontaneous complete remission was 3 years. Conclusions: Our study showed that almost one-third of patients with chronic ITP experienced spontaneous complete remission in an average of 3 years, and splenectomy provided satisfactory results in severe cases. This study demonstrates the natural history of chronic ITP in childhood before the era of eltrombopag. [ABSTRACT FROM AUTHOR]

Background: There is limited information on the natural course of chronic ITP in children. We aimed to evaluate the clinical and demographic characteristics of children with chronic ITP in the era before the availability of eltrombopag. Methods: A total of 86 children with chronic ITP between 1978–2014 were included. Demographic findings, laboratory results, clinical signs, bleeding scores, response time and time of complete remission were recorded. Results: The male/female ratio was 1.09, and median follow-up time was 3 years (range: 1.5–17 years). The median age at diagnosis of chronic ITP was 7 years (range: 2–17), and the median initial platelet count was 10 × 109/L (range: 1–66 × 109/L). Petechiae/ecchymoses were the most common clinical sign (86%) and followed by mucosal bleeding (39.5%). Severe bleeding was seen in 5% of the patients. None of them had intracranial hemorrhage. Twenty patients underwent splenectomy, and the rate of complete remission was 70%. Spontaneous complete remission was seen in 29% of the patients, and the median time to spontaneous complete remission was 3 years. Conclusions: Our study showed that almost one-third of patients with chronic ITP experienced spontaneous complete remission in an average of 3 years, and splenectomy provided satisfactory results in severe cases. This study demonstrates the natural history of chronic ITP in childhood before the era of eltrombopag.

Myosin heavy chain 9 (MYH9)-related platelet disorders (MYH9-RD) belong to the group of inherited thrombocytopenias characterized by giant platelets and Döhle bodies. The process leading to the diagnosis of MYH9-RD in a 13-year-old male patient, followed by the diagnosis of chronic immune thrombocytopenic purpura (ITP), is described. The patient had thrombocytopenia with increased mean platelet volume since he was a little boy. Low CD41, CD42 and CD61 levels were detected in blood tests sent to complete missing diagnostic tests. Platelet aggregation tests were also abnormal. The requested genetic test revealed a heterozygous mutation in the MYH9 gene. The patient's audiogram and kidney functions were normal. In conclusion, because MYH9-RD appears to be rare, it is of great importance to maintain a high index of suspicion when managing patients diagnosed with chronic ITP. Additional complaints and findings should be considered at every outpatient clinic examination to make a more accurate diagnosis and prevent unnecessary treatments. [ABSTRACT FROM AUTHOR]

Background : : This study aimed to identify chronicity predictors of pediatric primary immune thrombocytopenia (ITP). Methods : : This study retrospectively reviewed the medical records of patients with primary pediatric ITP admitted to a tertiary medical center between 2010 and 2021. Forty-five patients with a platelet count

Thrombopoietin receptor agonists are important therapeutic option in children with immune thrombocytopenia (ITP). We evaluated the response, efficacy, safety of a generic form of romiplostim manufactured in India for treating children with persistent/chronic ITP at our centre. Study of 45 children with persistent/chronic ITP was conducted of which 5 discontinued and 40 were included between 2019‐2020. Patients received romiplostim for 20 weeks, at a dose of 5 mcg/kg/week. Platelet count at week 1, 3, 20 and 26 was assessed. Predesigned algorithm was used for dose adjustment. After 20 weeks, patients who had platelet count of 50 × 109/L or above were tapered off medication and monitored till 26 weeks. Median platelet count at enrolment was 11 x 109/L (IQR 23 X 109/L). 13/40 children had received >/= three lines of prior ITP therapy. Platelet response (platelet count rise to more than 50×109/L without rescue medications) observed in 26 (65%) patients at week 20. Rescue medication was used in 12/40 children. Sustained platelet response after tapering and stopping romiplostim observed in 22/40 children. No adverse events were considered serious or led to discontinuation of treatment. Our data demonstrated generic romiplostim is well tolerated and efficacious in children with persistent/chronic ITP. [ABSTRACT FROM AUTHOR]

The present case series described six chronic immune thrombocytopenia patients (cITP), with a median age of 7.7 (7.0 to 13.0) years and low platelet count at 15,500 (7,000 to 20,000)/uL. They were suffering from bleeding symptoms and side effects of treatment. After enrollment, they were treated with thrombopoietin receptor agonist (eltrombopag). Five patients responded positively, showing a median platelet count of 115,000 (39,000 to 433,000)/uL. The median dose of eltrombopag used was 1.3 (0.8 to 2.2) mg/kg/day. The quality of life (QoL) improved for all patients, with their median overall score using a Pediatric QoL questionnaire showing 25.0% improvement. Median scores also showed improvements in each sphere of life functioning as physical (30.8%), emotional (26.4%), social (16.4%), and school (21.4%). The present report demonstrated that a select group of cITP patients, with low platelet count and bleeding symptoms, benefitted from treatment with eltrombopag, as shown by increased platelet counts and improved QoL. [ABSTRACT FROM AUTHOR]

Background: Chronic ITP is a very common disease in children. Most treatment options have focused on slowing platelet destruction like corticosteroids and IVIG. These treatments are not always effective, or have only a transient effect, and treatment related adverse events often restrict further use. Impaired platelet production has been evolved as a potential disease mechanism for ITP. As a result, growth factor and growth factor analogue stimulation of megakaryopoiesis has also been investigated. Objective: is to evaluate the efficacy and safety of eltrombopag for the treatment of children and adolescents with chronic ITP. Subjects and methods: It was a prospective cohort study which was conducted on forty patients (22 males and 18 females) with chronic ITP. They were treated with eltrombopag for a period ranged from 6 months to 2 years after failure of first line of treatment (steroids, IVIG or both). Patients started eltrombopag with mean initial platelet count 14.4 X 103/ uL and were evaluated after at least 6 months of therapy. The study was approved by the academic and medical ethics committee of Zagazig University and a written informed consent was obtained from all patients. Results: Thirty patients (75%) responded to eltrombopag with a mean platelet count of 100.9 X103/ uL while 10 patients (25%) did not respond to the drug. Mean platelet count was significantly higher after eltrombopag therapy (100.9 ± 65.97 versus 14.4 ± 8.87, P < 0.001). The longer the duration of treatment, the higher the mean platelet count after treatment (r = 0.4, P < 0.001). Only 43.3% of responders received combined treatment with IVIG and steroids versus 20% of non-responders (p = 0.006). Conclusion: Eltrombopag is an effective, well tolerated and safe drug to restore platelet counts at levels high enough to prevent bleeding events in children with chronic ITP. [ABSTRACT FROM AUTHOR]

To evaluate the rituximab treatment in children with chronic immune thrombocytopenic purpura This study included ten children with chronic immune thrombocytopenic purpura, which were nonresponsive to Steroid (S), IVIG and anti-D treatments. Rituximab was given with a dosage of 375 mg/m2 weekly for 4–6 weeks. Initial platelet count was less than 30×109/L and responses were assessed in follow-up. The patients’ groups were categorized as complete remission (CR);a platelet count ≥150×109/L,partial remission (PR);a platelet count ranging from 50×109/L to 150×109/L, minimal remission (MR); a platelet count ranging from 30×109/L to 50×109/L and no response (NR); a platelet count less than 30×109/L. Of our patients, four female and six male, their ages ranged from 39 mth to 13 yr and the mean age was 83.4±44.58 mth. None of the patients was splenectomized. The follow-up period after rituximab treatment ranged between 12 to 42 mth and the mean follow-up period was 25.10±13.03 months. While on this treatment, we had a CR in two patients, a PR in one, a MR in three, but no response in four. The patients in CR/PR are still being followed as in remission and they have 40 mth of mean follow-up period. The three patients in MR had a decrease in values of platelets earliest in one mth and the latest in four mth. Adverse effects of rituximab, such as itching and scraps that were not clinically significant were observed in three patients during rituximab infusion. There were no increase in infections after rituximab in any patient. CR was found in 20% of our patients, PR in 10% and MR in 30% with rituximab. On this treatment, while some series had good outcomes with this treatment (72%–100%, remission ratios), but many series, such as ours, had a poor response rate contrast to many reported case series in the literature. This condition may be associated with the age of our most patients who were young at the time of commenced rituximab. However, we believe that more studies are required to elucidate the reasons for different results in different case series reported in literature. [ABSTRACT FROM AUTHOR]