Diagnosis and Treatment of Myelofibrosis - Episode 2

Myelofibrosis: Goals of Therapy and Survival Outcomes

Jamile Shammo, MD:Goals of therapy in myelofibrosis evolve around a patient’s presentation. Exactly what are their symptoms, what is compromising their quality of life? In this situation, clearly, anemia is one item that we need to think about improving. We also want to improve symptoms of splenomegaly, as well as improving the symptoms of night sweats that the patient is dealing with because these tend to be relatively difficult to manage as you evolve with the disease. This is a relentless type of a problem that patients deal with. And so, I think management will be centered around improving the cytopenias, controlling splenomegaly, and improving the constitutional symptoms.

Patients who have low- to intermediate-risk disease tend to have better survival. Essentially, the IPSS risk categorization gave patients who have 0 score, which is considered low-risk, a median survival of around 11 years. So, you can contrast that with the high-risk disease case that we have talked about, and you can see the tremendous difference. For people who have intermediate-1 risk myelofibrosis, their median survival is on the order of 8 years. So, you can see how important it is to actually prognosticate as you make a diagnosis. And, importantly, the IPSS is something that we need to utilize at the time of diagnosis, and you cannot really redo it or reutilize it, say, 2 or 3 years later.

Myelofibrosis is not a very good disease to have. Taken altogether, people who have myelofibrosis have a median survival of about 6 years. Clearly, that has taken into account the low-risk and the high-risk disease, so that gives you the 6-year mark. Unfortunately, this is not a very common, and in order to be able to appreciate the variability from one patient to the next, you need to have a reasonable number of patients to kind of gain experience and get the flavor of each different case. But because the disease is not very common, I suspect that that may be one reason why appreciating the seriousness of this illness may not necessarily take place.

Transcript edited for clarity.

June 2016

  • A 72-year old male presents to his primary care physician with fatigue, abdominal pain lasting 3 months, and night sweats
  • PMH includes depression and gout
  • Physical Exam: BP, 155/85; spleen palpable 6 cm below costal margin
  • Laboratory values:
    • Hb= 9.8 g/L
    • HCT= 38%
    • WBC= 22.3 x109/L
    • Platelets= 255 x109/L
  • Bone marrow biopsy:
    • Megakaryocytic proliferation and atypia with evidence of reticulin fibrosis
  • Blood smear showed leukoerythroblastosis
  • Genetic testing showsJAK2 V617Fmutation
  • Diagnosis: Primary myelofibrosis