This is a prospective, randomized, open-label, active-controlled study to evaluate the efficacy and safety of Romiplostim N01 plus all-trans retinoic acid (ATRA) compared with Romiplostim N01 alone in adults with persistent isolated chemotherapy-induced thrombocytopenia (PICIT) after complete remission of gastrointestinal/digestive system solid tumors, including but not limited to gastrointestinal tract, pancreatic, and colorectal cancers.
Eligible participants will be randomized in a 1:1 ratio to receive Romiplostim N01 plus oral ATRA or Romiplostim N01 alone for 12 weeks, with follow-up through Week 24. The primary outcome is the overall platelet response rate at Week 12, defined as platelet count \>50 x 10\^9/L in at least 2 of the last 3 scheduled platelet assessments up to Week 12. Secondary outcomes include sustained response during Weeks 13 to 24, complete and partial response rates, duration of response, time to response, platelet count changes, platelet transfusion requirements, bleeding events, and safety.
Age 18 years or older.
Prior diagnosis of a gastrointestinal/digestive system solid tumor, including but not limited to gastrointestinal tract, pancreatic, or colorectal cancer.
Complete remission of the underlying tumor after chemotherapy or antitumor treatment, with tumor-related treatment discontinued for at least 12 weeks before enrollment, no evidence of recurrence or progression by specialist assessment, and no current need for additional tumor-directed therapy.
Persistent isolated chemotherapy-induced thrombocytopenia, defined as platelet count <30 x 10^9/L on two peripheral blood tests at least 7 days apart; or platelet count slightly higher than 30 x 10^9/L with dependence on platelet transfusion to maintain a safe platelet level.
Other hematologic diseases that may affect hematopoiesis or cause thrombocytopenia, including but not limited to aplastic anemia, myelodysplastic syndrome, leukemia or other hematologic malignancies, or a clear history of primary immune thrombocytopenia.
Active recurrence or progression of the underlying tumor, or evidence of bone marrow metastasis or tumor cell infiltration on bone marrow examination.
Uncontrolled chronic viral infection, including hepatitis B, hepatitis C, or HIV infection, or active severe infection at screening or within 4 weeks before screening.
Severe cardiac, hepatic, renal, or other organ dysfunction, or any serious organic disease that would make the participant unable to tolerate study treatment.
Parallel
Romiplostim N01
DrugRomiplostim N01 will be administered by subcutaneous injection at an initial dose of 4 mcg/kg once weekly for 12 weeks. The dose may be adjusted according to platelet count: increase by 2 mcg/kg if platelet count is \<50 x 10\^9/L, with a maximum dose of 10 mcg/kg; maintain the current dose if platelet count is \>=50 to \<=200 x 10\^9/L; reduce by 1 mcg/kg if platelet count is \>200 to \<=400 x 10\^9/L; and withhold dosing if platelet count is \>400 x 10\^9/L, then restart at a lower dose after platelet count decreases to approximately 200 x 10\^9/L.
All-trans retinoic acid
DrugAll-trans retinoic acid (ATRA) will be administered orally at 10 mg twice daily for 12 weeks. Dose interruption, reduction, or discontinuation may be performed for intolerable toxicity or clinically significant adverse events according to the protocol.
Overall Platelet Response Rate at Week 12
Percentage of participants with platelet count \>50 x 10\^9/L in at least 2 of the last 3 scheduled platelet assessments up to Week 12. Platelet transfusion or other rescue/supportive treatment will not be counted as a platelet response.
Sustained Platelet Response Rate During Weeks 13 to 24
Percentage of participants with platelet count \>50 x 10\^9/L in at least 2 of the last 3 scheduled platelet assessments during Weeks 13 to 24 and without rescue therapy during this period.
Complete Response Rate
Percentage of participants with platelet count \>=100 x 10\^9/L and no bleeding for at least 2 consecutive weeks.
Partial Response Rate
Percentage of participants with platelet count \>=30 x 10\^9/L and at least 2 times the baseline platelet count, with no bleeding.
Duration of Response
Among responders, time from first achievement of platelet response to platelet count \<30 x 10\^9/L or the end of study follow-up.
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Peking University People's Hospital
Lead sponsor
Peking University Cancer Hospital and Institute
Collaborator
Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Collaborator
Peking University International Hospital
Collaborator
Shanxi Province Cancer Hospital
Collaborator
Henan Provincial People's Hospital
Collaborator
China-Japan Union Hospital, Sun Yat-sen University Cancer Center
Collaborator
Hebei Medical University Fourth Hospital
Collaborator
Tianjin Medical University Cancer Institute and Hospital
Collaborator
