Use of pegylated interferon in young patients with polycythemia vera and essential thrombocythemia

Abstract

Myeloproliferative neoplasms (MPN) are rare disorders in young patients, and because of this, standardized treatment recommendations are not available. Pediatric patients are more frequently treated with hydroxyurea than interferon, yet there are no data suggesting this is the best practice. Current treatment guidelines for adults suggest using interferon as upfront therapy in young patients. We reviewed the cases of 13 young patients with polycythemia vera or essential thrombocythemia, who were treated with interferon. Extreme thrombocytosis was well controlled and the medication was tolerated by many. Our work shows the need for prospective studies evaluating interferon in our youngest patients with MPN.

Position paper: The time for cooperative group study of ropeginterferon alfa‐2b in young patients with myeloproliferative neoplasms is now

“…They do not share the controversy surrounding hydroxyurea's potential for mutagenicity, 14 and there has been data showing that use of IFN in some pregnant MPN patients led to increased odds of live births 18 . IFN has been used previously in many children and teenagers 8,19 . Our group observed favorable outcomes with IFN in a small series of pediatric patients with MPNs (six with ET and seven with PV, of whom two‐thirds harbored a JAK2 mutation), including normalization of platelet counts and decrease in, or elimination of, phlebotomy 19 …”

“…18 IFN has been used previously in many children and teenagers. 8,19 Our group observed favorable outcomes with IFN in a small series of pediatric patients with MPNs (six with ET and seven with PV, of whom two-thirds harbored a JAK2 mutation), including normalization of platelet counts and decrease in, or elimination of, phlebotomy. 19 Side effects that may limit IFN's use in some patients include the potential for psychiatric or autoimmune effects, and elevations in liver enzymes.…”

“…8,19 Our group observed favorable outcomes with IFN in a small series of pediatric patients with MPNs (six with ET and seven with PV, of whom two-thirds harbored a JAK2 mutation), including normalization of platelet counts and decrease in, or elimination of, phlebotomy. 19 Side effects that may limit IFN's use in some patients include the potential for psychiatric or autoimmune effects, and elevations in liver enzymes. 19,20 In our prior study, five of 13 children discontinued IFN-2 for mood related effects, one for transaminitis post-liver transplant, and two because of VTE events that occurred while on IFN 19 (although one of those patients then restarted IFN soon after and remained on it without additional VTE events or side effects).…”

“…19 Side effects that may limit IFN's use in some patients include the potential for psychiatric or autoimmune effects, and elevations in liver enzymes. 19,20 In our prior study, five of 13 children discontinued IFN-2 for mood related effects, one for transaminitis post-liver transplant, and two because of VTE events that occurred while on IFN 19 (although one of those patients then restarted IFN soon after and remained on it without additional VTE events or side effects).…”

“…Milder side effects that did not require discontinuation included dizziness and injection site reaction. 19 Since the publication of that cohort, we have treated additional young patients with IFN who have had improvement in, or normalization of, counts and elimination of phlebotomies with resolution of iron deficiency. Side effects have ranged from asymptomatic to brief periods of fatigue or nausea.…”