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The objective of this observational study is to explore the long-term effects of roprastine given to promote platelet implantation in hematopoietic stem cell hemicongruent transplantation in children with thalassemia. The main questions it aimed to answer is:
Is roprastine safe and effective for platelet implantation in children with thalassemia hemicongruent transplantation? Participants who have already received roprastine as part of routine medical care for hematopoietic stem cell hemicongruent transplantation in children with thalassemia will answer online survey questions about the effects of their platelet implantation within 8 weeks.
Allogeneic hematopoietic stem cell transplantation is an important, if not the only, means of curing many diseases of the blood system. Thrombocytopenia after transplantation seriously affects the long-term survival rate of patients. allo - The incidence of thrombocytopenia in HSCT patients is 5-20% (< 20 x 10 ^ 9/L), increasing the risk and cost of treatment. There are currently few studies on the promotion of platelet growth in children with thalassemia. Repeated infusion of platelet suspension can lead to many adverse consequences, including blood transfusion reactions, platelet homeoimmune responses, and transfusion-associated virus infections. There is a black box warning of liver toxicity in eltropopa, and the incidence of real-world liver toxicity is 11.8%. Transplant patients are prone to diarrhea, which affects the absorption of oral platelet-raising drugs. Daily subcutaneous injection increases children's pain and poor tolerance. However, the long-acting platelet-raising drugs once after transplantation are well tolerated in children with thalassemia transplantation, and the efficacy is worth looking forward to. Up to now, there is a lack of relevant clinical data on the application of roprastine to promote platelet recovery in children with thalassemia hemiphase transplantation. Therefore, this study aimed to explore the observational study of roprastine to promote platelet implantation in children with thalassemia hemiphase transplantation, and to explore the efficacy and safety of the drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| experimental group | After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Romiplostim | Drug | After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective. |
| Measure | Description | Time Frame |
|---|---|---|
| Platelet recovery time | The time at nodes such as platelet >20×10^9/L, 50×10^9/L and 100×10^9/L | From enrollment to 28 days after transplantation |
| Platelet transfusion volume | The required dosage of platelet suspension | From enrollment to 28 days after transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse drug reaction rate | The rate of adverse drug reactions occurring during the follow-up period | From enrollment to 28 days after transplantation |
| Bleeding incidence rate | The incidence rate of bleeding that occurred during the follow-up period |
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Inclusion Criteria:
Exclusion Criteria:
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Children aged 2 - 17 years old who were diagnosed with severe thalassemia at Haikou People's Hospital and received haploidentical hematopoietic stem cell transplantation.
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| Name | Affiliation | Role |
|---|---|---|
| Xiaoyang Yang, MD | Department of Hematology, Haikou People's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Haikou Affiliated Hospital of Central South University Xiangya School of Medicine | Haikou | Hainan | 570208 | China |
The IPD encompasses highly sensitive and confidential data that was collected through significant investment of our resources, both in terms of time and funding. This data is integral to our ongoing research initiatives, which are at a crucial stage of development. Premature sharing could disrupt our research timelines and strategic plans. Additionally, we have not yet established comprehensive safeguards to ensure that the data will be used appropriately by external researchers. Without proper protocols in place, there is a risk of misuse or misinterpretation of the data, which could lead to inaccurate research outcomes and potential reputational damage to our institution. For these reasons, we have decided not to share the IPD at this time.
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| ID | Term |
|---|---|
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C488777 | romiplostim |
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|
|
| From enrollment to 28 days after transplantation |
| Thrombosis incidence rate | The incidence rate of thrombosis occurring during the follow-up period | From enrollment to 28 days after transplantation |
| Survival rate | The survival rate of patients after transplantation medication | From enrollment to 28 days after transplantation |
| Recurrence - free survival rate | The survival rate of patients without recurrence of the disease after using the drug | From enrollment to 28 days after transplantation |
| Transplant - related mortality | From enrollment to 28 days after transplantation |
| Major transplant-related complications | From enrollment to 28 days after transplantation |
| Cause of death | From enrollment to 28 days after transplantation |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |