Myeloproliferative neoplasms are chronic blood cancers in which the bone marrow produces too many blood cells. Patients with Philadelphia chromosome-negative myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia, and primary myelofibrosis, may need treatment to reduce high blood cell counts, relieve disease-related symptoms, and lower the risk of complications. However, currently available cytoreductive treatments may be ineffective, poorly tolerated, or inconvenient for some patients.
Selinexor is an oral selective inhibitor of nuclear export that has shown antitumor activity in several hematologic malignancies. This study will evaluate the effectiveness and safety of selinexor used alone as cytoreductive treatment in patients with Philadelphia chromosome-negative myeloproliferative neoplasms who have an indication for cytoreductive therapy.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral selinexor, with dose adjustments based on tolerability and blood cell counts. Participants will be followed for treatment response, symptom improvement, and side effects for up to 6 months. The results of this study may help determine whether selinexor could provide a potential treatment option for patients with Philadelphia chromosome-negative myeloproliferative neoplasms who have limited cytoreductive therapy choices.
Aged 18 to 80 years at the time of informed consent.
Diagnosis of Philadelphia chromosome-negative myeloproliferative neoplasm, including polycythemia vera, essential thrombocythemia, or primary myelofibrosis, according to World Health Organization criteria.
Extreme thrombocytosis, defined as platelet count >1500 x 10^9/L.
Progressive leukocytosis, defined as white blood cell count >25 x 10^9/L.
Diagnosis of Philadelphia chromosome-positive myeloproliferative neoplasm or chronic myeloid leukemia.
Concurrent acute leukemia or other active hematologic malignancy.
Severe or uncontrolled comorbid condition, including but not limited to significant cardiac or pulmonary disease, decompensated liver disease, or end-stage renal disease.
Active uncontrolled bacterial, viral, or fungal infection.
Single group
Selinexor
DrugSelinexor will be administered orally at an initial dose of 40 mg once weekly. Treatment may be continued for up to 3 months, with a planned total follow-up period of 6 months. Dose escalation to 60 mg or 80 mg once weekly is permitted in participants without significant hematologic or non-hematologic toxicity, according to the investigator's judgment. Dose interruption and dose reduction are allowed based on safety and tolerability. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, selinexor will be temporarily withheld and resumed at a reduced dose after recovery. Treatment will be permanently discontinued if unacceptable toxicity persists despite dose modification.
Proportion of Participants Achieving Disease-Relevant Peripheral Blood Count Control
Disease-relevant peripheral blood count control will be assessed using hematologic parameters, including platelet count, white blood cell count, and hematocrit where applicable. A participant will be considered to have achieved cytoreductive response if the elevated disease-relevant blood count parameter at baseline normalizes or shows a clinically meaningful reduction from baseline according to the study protocol.
Incidence of Treatment-Emergent Adverse Events
Treatment-emergent adverse events will be assessed according to clinical evaluation and laboratory testing during the study period.
Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
Change From Baseline in Disease-Related Symptom Burden Assessed by the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score
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