| Case 1 |
| A 68-year-old female was initially diagnosed with polycythemia vera in 2001, and initially treated with intermittent phlebotomy, low dose aspirin, and hydroxyurea. Her need for phlebotomy decreased in 2008 and was no longer required after 2009. In January 2012, she presented with abdominal fullness, decreased appetite, marked fatigue and a weight loss of 10 kg. |
| Her ECOG performance score was 2; the spleen was palpable by 20 cm below the left costal margin. The Hgb was 96g/L, WBC 26 × 109/L, and platelets 300 × 109/L. The differential count showed left shift, nucleated red cells and 2% myeloblasts. Her LDH was 985U/L (IULN 240). |
| A bone marrow biopsy showed grade 3/3 fibrosis, <5% myeloblasts, and normal cytogenetics. Cells expressed the JAK2V617F mutation. The patient has two healthy siblings, ages 64 and 66 years. |
Question 1: How would you treat this patient?
Wait and watch Continue hydroxyurea Immediate allogeneic transplant if a suitable donor identified JAK1/2 inhibitor therapy with transplantation only if JAK inhibitor therapy fails JAK1/2 inhibitor therapy with transplantation as soon as spleen size has been reduced
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Discussion: The patient’s physician started her on the JAK1/2 inhibitor ruxolitinib, and after 6 weeks, she had significant improvement in symptoms. Her blood counts were stable, and she was transfusion independent. HLA typing, meanwhile, shows one of her siblings to be HLA-identical. |
Question 2: What would you recommend at this point?
Proceed with allogeneic transplantation from her sibling donor Continue ruxolitinib; no need for transplantation. Other options
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Discussion: This patient had a gratifying response to JAK1/2 inhibition. As a 68-year-old patient, transplantation would, presumably, involve an RIC regimen. While some data suggest a higher probability of relapse with RIC than observed with high intensity conditioning, other reports have shown low relapse rates. While there is a 50–60% probability of long-term survival and remission, there is also approximately a 50% chance of developing GVHD requiring therapy, possibly long-term. Thus, with the present state of knowledge, it is difficult to provide an absolutely definitive recommendation. Some patients might prefer conservative management with continuation of ruxolitinib until there are signs of disease acceleration/progression. Other patients (and physicians) might prefer to proceed with HCT, acknowledging the risk of GVHD, but valuing the potential for cure higher than the possibility of relapse and risk of GVHD. |
