Nplate Can Cause Bone Marrow Fibrosis

In a clinical study of nplate involving 271 patients, four patients had to discontinue treatment due to the formation of reticulin fiber deposition within the bone marrow. Six more patients were found to have reticulin progression after having a bone marrow biopsy.

Reticulin fiber deposition is the early stage of bone marrow fibrosis, also known as myelofibrosis. This is a bone disorder where fibrous scar tissue replaces bone marrow, inhibiting the patient from developing new blood cells. As fewer blood cells are generated, less platelets as well as red and white blood cells are able to form (a condition called pancytopenia). Symptoms include bone pain, bruising and easy bleeding, fatigue, shortness of breath due to anemia, and abdominal fullness caused by an enlarged spleen.

Although all the patients in the controlled studies were able to stop treatment of Nplate before their reticulin progressed to bone marrow fibrosis, one patient in an extension study did develop fibrosis. As this happens, the patients may experience very low blood cell counts.

What do you think? Nplate is a new drug with potentially unknown long term effects. Please share any opinions or experiences you have concerning Nplate.