Thrombocytopenia secondary to hematopoietic stem cell transplantation. Thrombocytopenia, unspecified
Conditions
Interventions
Intervention group: Patients will receive treatment with Romiplostim (brand name Nplate, manufactured by Amgen, USA). The drug will be administered at a dose of 5 micrograms per kilogram of body weigh
Sponsors
Tehran University of Medical Sciences
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: Age 18 years or less Both genders Patients who have received hematopoietic stem cell transplantation (either allogeneic or autologous) Post-HSCT thrombocytopenia, defined as:1- PIT (Prolonged Isolated Thrombocytopenia): Failure of platelet count to rise above 20 × 10?/L or clinical dependence on platelet transfusion for more than 60 days after HSCT. 2- SFPR (Secondary Failure of Platelet Recovery): Platelet count 20 × 10?/L for 7 days without transfusion, indicating initial engraftment. Normal organ function for at least 7 days prior to enrollment, defined as: 1- Acceptable pulmonary function. 2- Serum creatinine < 1.5 times the upper normal limit for age (with GFR calculation). 3- Acceptable liver function, including: Elevated liver enzymes acceptable unless there is evidence of chronic liver disease,Total bilirubin = 3 × upper normal limit, Conjugated bilirubin < 2 mg/dL. 4- Normal cardiac function Estimated life expectancy of at least 1 year Written informed consent obtained from the patient or legal guardian
Exclusion criteria
Exclusion criteria: Presence of underlying conditions or disorders predisposing the patient to thrombosis Patients with a history of complex karyotype or mutations such as monosomy 7 or 5q deletion prior to transplantation, or other cytogenetic abnormalities associated with a predisposition to malignancy. Previous or new diagnosis of myelodysplastic syndrome (MDS) Use of any investigational drug within 30 days prior to enrollment Uncontrolled active infection (e.g., sepsis, hepatitis B, or hepatitis C), and patients with HIV Chronic liver disease (e.g., cirrhosis or fibrosis) Splenomegaly Known hypersensitivity to Romiplostim-related chemical compounds or E. coli–derived products that contraindicates the use of Romiplostim
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of patients who achieve platelet count above 100×10?/L after treatment (Complete response). Timepoint: Weekly for 12 weeks after initiation of treatment; then every other week for 12 weeks. Method of measurement: Complete blood count test.;Number of patients who achieve platelet count above 50×10?/L after treatment (Complete response). Timepoint: Weekly for 12 weeks after initiation of treatment; then every other week for 12 weeks. Method of measurement: Complete blood count.;Number of patients with an increase in platelet count >10×10?/L from baseline (minimal response). Timepoint: Weekly for 12 weeks after initiation of treatment; then every other week for 12 weeks. Method of measurement: Complete blood count.;Number of patients with no response (platelet count increase <10×10?/L from baseline). Timepoint: Weekly for 12 weeks after initiation of treatment; then every other week for 12 weeks. Method of measurement: Complete blood count. | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in hemoglobin level before and after Romiplostim treatment. Timepoint: Weekly assessments from 4 weeks before treatment initiation until 12 weeks after, followed by biweekly assessments until week 24. Method of measurement: Complete blood count test.;Change in absolute neutrophil count before and after Romiplostim treatment. Timepoint: Weekly assessments from 4 weeks before treatment initiation until 12 weeks after, followed by biweekly assessments until week 24. Method of measurement: Complete blood count test.;Number of packed red blood cell transfusions before and after Romiplostim initiation. Timepoint: Weekly, 8 weeks before and 24 weeks after initiation of treatment. Method of measurement: patient medical records and hospital blood bank documentation.".;Number of platelet transfusions before and after Romiplostim initiation. Timepoint: Weekly, 8 weeks before and 24 weeks after initiation of treatment. Method of measurement: patient medical records and hospital blood bank documentation.".;Incidence of treatment-related adverse events. Timepoint: Weekly, for 24 weeks after initiation of treatment. Method of measurement: National Cancer Institute – Common Terminology Criteria for Adverse Events, version 5.0.;Incidence of bleeding events. Timepoint: Weekly, for 24 weeks after initiation of treatment. Method of measurement: WHO bleeding scale.;Change in platelet count before and after Romiplostim treatment. Timepoint: Weekly assessments from 4 weeks before treatment initiation until 12 weeks after, followed by biweekly assessments until week 24. Method of measurement: Complete blood count test. | — |
Countries
Isle of Man
Contacts
Public ContactAmir Ali Hamidieh
Tehran University of Medical Sciences
Outcome results
None listed