76 year old male had not seen a doctor in many years. Presented with constitutional symptoms and pancytopenia, most notably severe thrombocytopenia. No significant splenomegaly. Bone marrow biopsy revealed marked fibrosis, megakaryocytic dysplasia, 2% blasts, del20q, trisomy 21, JAK2, TET2, U2AF1. We tried him for a few months on Vonjo, everything is basically unchanged, he still has severe thrombocytopenia. His functional status is borderline for a transplant consult. Hgb and white count are low but not severely. Any suggestions to help with the thrombocytopenia?
Unfortunately, it looks like this patient has very high risk disease. Trisomy 21 is associated with high risk of progression to AML (blast phase). U2AF1 often associated with anemia/thrombocytopenia. While pacritinib is approved for myelofibrosis with markedly low platelets, it does not increase platelet count. Rather, it can be safely used to improve splenomegaly / symptoms in patients with low platelets since it doesn't worsen platelet counts. Is the patient transfusion dependent on platelets? Usually, there isn't much clinical benefit in raising platelets unless pt are having issues with bleeding or if they are requiring transfusions. If you absolutely need to raise platelet counts, then would consider danazol or thalidomide/prednisone. Other option would be using hypomethylating agent (will worsen platelet count before it helps). Ultimately, this pt is likely to progress to accelerated/blast phase disease and you'll have to use hypomethylating agent at that point as well.
