Pediatric Polycythemia Treatment & Management
- Author: Joseph K Park, MD, PhD; Chief Editor: Max J Coppes, MD, PhD, MBA more...
The goals of therapy are to maximize survival while minimizing the complications of therapy as well as of the disease itself. Phlebotomy and myelosuppressive chemotherapy are the cornerstones of therapy. Based on clinical trials in adults, these approaches have produced a median survival time of 9-14 years after the beginning of treatment. Current recommendations for treatment of young patients primarily rely on phlebotomy because the thrombosis is far less likely to occur in children and the long-term risks of leukemia over a longer life span are increased.
The goal of phlebotomy is to maintain normal red cell mass and blood volume, with a target hematocrit level of 42-46% for men and 39-42% for women. The mean survival time of adult patients treated solely with phlebotomy is 13.9 years; however, a high risk of thromboembolic complications is observed. To reduce the thrombotic risk, antiplatelet agents such as aspirin and dipyridamole were used in combination with phlebotomy. Initial studies demonstrated an increased risk of hemorrhage in the phlebotomy plus aspirin/dipyridamole arm. However, a large European study showed a decrease in thrombotic events in those patients receiving low-dose aspirin therapy and recommended aspirin therapy for those patients for whom no contraindications were noted.
Hydroxyurea as a myelosuppressive agent is also widely used in high-risk patients with polycythemia vera (ie, >60 y, history of thrombosis) who require cytoreductive therapy, reducing the need for phlebotomy. Neutropenia and thrombocytopenia are expected adverse effects, both of which can be rapidly corrected after holding or reducing the medication dose. The incidence of thrombotic complications is less than compared with patients treated with phlebotomy alone. However, these patients also experience higher rates of malignancy. Historically, patients have been treated with chlorambucil and busulfan. However, these patients exhibited the highest rates of secondary malignancy including acute leukemia, lymphocytic lymphomas, and skin and GI carcinomas. The rates of malignancy appear lower with busulfan than with the other alkylating agents. Currently, these agents are rarely used.
Patients treated with phosphorus-32 (32 P) tolerate treatment well and have prolonged periods of remission. However, these patients also exhibit increased rates of acute leukemias (10-15%). The mean survival time with32 P treatment is 10.9 years; therefore, phosphorous is rarely used.
Interferon-alpha is effective in eliminating J AK2V617Fexpression and inducing hematologic remission. Its use is limited by side effects, cost, and route of administration. The pegylated form and low dose treatment has decreased the rate of discontinuation of the drug secondary to side effects. In a French study, patients with polycythemia vera treated with interferon alpha showed a high rate of hematologic and molecular response.[33, 34]
Tyrosine kinase inhibitors
Imatinib is a BCR-ABL tyrosine kinase inhibitor, which has been used in some patients with polycythemia vera. It has been shown to decreased blood counts, splenomegaly, and need for phlebotomy.[35, 36, 37]
Ruxolitinib is a JAK1/JAK2 inhibitor was initially approved for primary myelofibrosis, and was used in a phase II trial of patients with PV who were refractory to hydroxyurea therapy. In this study, reduction in hematocrit, thrombocytosis, leukocytosis, and splenomegaly was seen in the ruxolitinib group.
Phlebotomy is used for symptomatic hyperviscosity. The goal is to treat the underlying cause of polycythemia.
See the list below:
Surgery is not typically indicated. Occasionally, splenectomy is performed late in the course of the disease if massive splenomegaly causes adverse effects such as early satiety, anemia, or thrombocytopenia from sequestration.
Please note that these patients have a high risk of complications during surgical procedures.
See the list below:
Consult a neurologist and neurosurgeon if evidence of a stroke is present.
See the list below:
Diet is unrestricted.
See the list below:
Contact sports and other activities should be limited for individuals in hypercoagulable and hypocoagulable states.
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