Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence

Inclusion Criteria:

Age ≥ 18 years
Myeloproliferative-neoplasm (MPN)-associated myelofibrosis

RBC-transfusion-dependence (global study):

Average RBC-transfusion frequency ≥ 2 units/28 days over at least the 84 days immediately prior to randomization. There must be no interval > 42 days without ≥ 1 RBC-transfusion.
Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in

determining eligibility.

RBC-transfusions due to bleeding are not scored in determining eligibility.

RBC-transfusions due to chemotherapy-induced anemia are not scored in determining eligibility.

Severe anemia (China-specific extension):
≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment.

No RBC-transfusion within 6 months prior to enrollment.

Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in the China-specific extension.
Bone marrow biopsy within 6 months (global study only).
Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
Agree to follow pregnancy precautions as required by the protocol.
Agree to receive counseling related to teratogenic and other risks of pomalidomide.
Agree not to donate blood or semen.

Exclusion Criteria:

Prior blood cell or bone marrow allotransplant.
Use of drugs to treat MPN-associated myelofibrosis ≤ 30 days before starting study drug.
Treatment with erythropoietin or androgenic steroids ≤ 84 days before starting study drug.
Anemia due to reasons other than MPN-associated myelofibrosis.
Pregnant or lactating females.
More than 10% blasts by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks

Prior history of malignancies,other than the disease being studied, unless the subject has been free of the malignancy for ≥ 5 years with the following exceptions:

Carcinoma in situ of the cervix
Carcinoma in situ of the breast
Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system)
Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections.
Prior treatment with pomalidomide.
Allergic reaction or rash after treatment with thalidomide or lenalidomide

Any of the following laboratory abnormalities:

Neutrophils < 0.5x10^9 /L
Platelets < 25 x 10^9 /L
Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m²
Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN)
Total bilirubin ≥ 4 x ULN;
Uncontrolled hyperthyroidism or hypothyroidism.
Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting study drug
Clinically-important heart disease within the past 6 months