Early Prophylactic rhTPO Administration for Thrombocytopenia Associated With EAP Regimen-Based Hematopoietic Stem Cell Mobilization

NCT07848672 — Phase 2 / Phase 3, Not yet recruiting, 70 participants, The Affiliated People's Hospital of Ningbo University

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A Single-Arm, Multicenter, Prospective Phase II Clinical Study of Early Prophylactic rhTPO for the Prevention of Hematopoietic Stem Cell Mobilization-Related Thrombocytopenia During the EAP Regimen

Registry	NCT07848672
Status	Not yet recruiting
Phase	Phase 2 / Phase 3
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	70 (Estimated)
Sponsor	The Affiliated People's Hospital of Ningbo University (Other gov)
Conditions	Multiple Myeloma (MM), Lymphoma, Large B-Cell, Diffuse (DLBCL), Lymphoma, Hematopoietic Stem Cell Mobilization, Chemotherapy-Induced Thrombocytopenia
Interventions	rhTPO prophylaxis plus EAP stem-cell mobilization (Drug)
Dates	start 2027-01-01 (estimated), primary completion 2029-12-31 (estimated), completion 2030-12-01 (estimated), first posted 2026-09-30, last updated 2026-09-30

-Summary

This is a multicenter, prospective, single-arm, phase II clinical study evaluating the early prophylactic use of recombinant human thrombopoietin (rhTPO) to prevent thrombocytopenia associated with the EAP regimen for hematopoietic stem cell mobilization. The EAP regimen consists of etoposide, cytarabine, and pegylated granulocyte colony-stimulating factor (pegfilgrastim) and is used for hematopoietic stem cell mobilization before autologous stem cell transplantation in patients with multiple myeloma and B-cell lymphoma.

Severe thrombocytopenia is a common complication during EAP-based stem cell mobilization and may increase the risk of bleeding and the need for platelet transfusions, and may sometimes delay stem cell collection. In this study, patients whose platelet counts decrease to ≤80 × 10⁹/L after EAP chemotherapy will receive rhTPO early as a preventive treatment. The study will evaluate whether early rhTPO administration can reduce the incidence of clinically significant bleeding and the need for platelet transfusion while maintaining adequate hematopoietic stem cell mobilization.

The study will also monitor platelet counts, stem cell collection outcomes, and treatment-related adverse events to evaluate the effectiveness and safety of early rhTPO administration.

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+Arms and interventions

Arm	Type	Interventions	Description
Prophylactic early-use rhTPO for chemotherapy-induced thrombocytopenia during EAP-based hematopoieti	Experimental	Drug: rhTPO prophylaxis plus EAP stem-cell mobilization	This is the only interventional arm of this multicenter, prospective, single-arm phase II study. Eligible patients with lymphoma or multiple myeloma receive standard EAP mobilization chemotherapy (etoposide 75 mg/m² i.v. d1-2, cytarabine 200 mg/m² q12h i.v. d1-2; pegfilgrastim is administered subcutaneously on day 6 after completion of chemotherapy; dose reduction to 2/3-3/4 of original dose is allowed for patients > 65 years or creatinine clearance 30-60 mL/min). After EAP chemotherapy, blood routine tests are monitored dynamically. When peripheral platelet count drops to ≤ 80 × 10⁹/L, prophylactic recombinant human thrombopoietin (rhTPO) 300 U/kg is given subcutaneously once daily. rhTPO administration continues until completion of stem-cell apheresis, platelet recovery > 50 × 10⁹/L, or investigator-judged discontinuation. Platelet transfusion is only permitted when platelet count < 20 × 10⁹/L or active bleeding occurs. Subjects undergo serial laboratory assessments including

rhTPO prophylaxis plus EAP stem-cell mobilization (Drug): Standard EAP mobilization chemotherapy (etoposide + cytarabine + pegfilgrastim) plus early prophylactic subcutaneous rhTPO 300 U/kg/day initiated when platelet ≤80 ×10⁹/L. rhTPO stops after stem-cell collection, platelet recovery >50 ×10⁹/L or investigator's decision. Platelet transfusion follows unified trigger criteria: PLT<20×10⁹/L or active bleeding.

+Outcome measures

Primary outcomes

Platelet transfusion rate during mobilization period [From initiation of EAP chemotherapy to completion of the last stem-cell apheresis,assessed up to 30 days.]: Proportion of participants receiving at least one platelet transfusion in the mobilization phase.
Incidence of grade ≥2 bleeding events during mobilization period [From initiation of EAP chemotherapy to completion of the last stem-cell apheresis,assessed up to 40 days.]: Proportion of participants experiencing CTCAE v5.0 grade ≥2 bleeding events throughout the stem-cell mobilization cycle.

Secondary outcomes

Nadir platelet count [From initiation of EAP chemotherapy to completion of the last stem-cell apheresis, assessed up to 40 days.]: The minimum peripheral platelet count observed in mobilization cycle.
Duration of platelet count <20 ×10⁹/L [From initiation of EAP chemotherapy to completion of the last stem-cell apheresis,assessed up to 40 days.]: Number of days with platelet count below 20 ×10⁹/L.
Proportion of participants with delayed stem-cell apheresis due to severe thrombocytopenia [From initiation of EAP chemotherapy to completion of the last stem-cell apheresis,assessed up to 30 days.]: Percentage of subjects whose apheresis is postponed because of severe thrombocytopenia.
Stem-cell mobilization success rate [At completion of stem-cell apheresis,assessed up to 30 days.]: Proportion of participants achieving total CD34⁺ cell yield ≥5 ×10⁶/kg body weight.
Incidence of adverse events [From start of study intervention to completion of stem-cell collection, assessed up to 40 days.]: Incidence of thromboembolism, liver injury, severe bone pain, severe infection graded by CTCAE v5.0.
Length of hospital stay during mobilization [From admission for EAP mobilization to discharge after apheresis finished,assessed up to 30 days.]: Total hospital days in the mobilization stage.

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

1. Age between 18 and 75 years old.
2. Confirmed diagnosis of multiple myeloma or B-cell lymphoma, eligible for autologous hematopoietic stem-cell transplantation and planned to receive EAP-based stem-cell mobilization.
3. Baseline peripheral platelet count ≥ 80 × 10⁹/L.
4. Adequate cardiac, hepatic and renal function to tolerate EAP-combination chemotherapy.
5. Willing and able to complete the full-study follow-up procedures.
6. Voluntarily provide written informed consent.

Exclusion Criteria:

1. Previous history of arterial or venous thromboembolism including pulmonary embolism.
2. Liver cirrhosis or baseline alanine transaminase (ALT) > 2 times upper limit of normal.
3. Active uncontrolled infection.
4. Confirmed myelofibrosis.
5. Pregnant or lactating female subjects.
6. Known hypersensitivity to recombinant human thrombopoietin (rhTPO).