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Pediatric Polycythemia Treatment & Management

  • Author: Joseph K Park, MD, PhD; Chief Editor: Max J Coppes, MD, PhD, MBA  more...
 
Updated: Apr 07, 2016
 

Medical Care

Primary polycythemia

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.

Phlebotomy

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.[31]

Hydroxyurea

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.[32] 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

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.[38]

Secondary polycythemia

Phlebotomy is used for symptomatic hyperviscosity. The goal is to treat the underlying cause of polycythemia.

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Surgical Care

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.
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Consultations

See the list below:

  • Consult a neurologist and neurosurgeon if evidence of a stroke is present.
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Diet

See the list below:

  • Diet is unrestricted.
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Activity

See the list below:

  • Contact sports and other activities should be limited for individuals in hypercoagulable and hypocoagulable states.
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Contributor Information and Disclosures
Author

Joseph K Park, MD, PhD Paul and Yuanbi Ramsay Endowed Postdoctoral Fellow, Division of Hematology/Oncology, Lucile Packard Children's Hospital at Stanford

Disclosure: Nothing to disclose.

Coauthor(s)

Kathleen M Sakamoto, MD, PhD Shelagh Galligan Professor, Division of Hematology/Oncology, Department of Pediatrics, Stanford University School of Medicine

Kathleen M Sakamoto, MD, PhD is a member of the following medical societies: International Society for Experimental Hematology, American Society of Hematology, American Society of Pediatric Hematology/Oncology, Society for Pediatric Research

Disclosure: Nothing to disclose.

Krysta D Schlis, MD Clinical Assistant Professor of Pediatrics, Lucile Packard Children’s Hospital, Stanford University School of Medicine

Krysta D Schlis, MD is a member of the following medical societies: American Society of Hematology, American Society of Pediatric Hematology/Oncology

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

James L Harper, MD Associate Professor, Department of Pediatrics, Division of Hematology/Oncology and Bone Marrow Transplantation, Associate Chairman for Education, Department of Pediatrics, University of Nebraska Medical Center; Associate Clinical Professor, Department of Pediatrics, Creighton University School of Medicine; Director, Continuing Medical Education, Children's Memorial Hospital; Pediatric Director, Nebraska Regional Hemophilia Treatment Center

James L Harper, MD is a member of the following medical societies: American Society of Pediatric Hematology/Oncology, American Federation for Clinical Research, Council on Medical Student Education in Pediatrics, Hemophilia and Thrombosis Research Society, American Academy of Pediatrics, American Association for Cancer Research, American Society of Hematology

Disclosure: Nothing to disclose.

Chief Editor

Max J Coppes, MD, PhD, MBA Executive Vice President, Chief Medical and Academic Officer, Renown Heath

Max J Coppes, MD, PhD, MBA is a member of the following medical societies: American College of Healthcare Executives, American Society of Pediatric Hematology/Oncology, Society for Pediatric Research

Disclosure: Nothing to disclose.

Additional Contributors

Scott S MacGilvray, MD Clinical Professor, Department of Pediatrics, Division of Neonatology, The Brody School of Medicine at East Carolina University

Scott S MacGilvray, MD is a member of the following medical societies: American Academy of Pediatrics

Disclosure: Nothing to disclose.

Acknowledgements

Kristin Baird, MD Staff Clinician, Pediatric Oncology Branch, National Cancer Institute, National Institutes of Health

Disclosure: Nothing to disclose.

Sun H Choo, MD Resident Physician, Department of Pediatrics, University of California, Los Angeles, David Geffen School of Medicine

Disclosure: Nothing to disclose.

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Bone marrow film at 400X magnification demonstrating dominance of erythropoiesis. Courtesy of U. Woermann, MD, Division of Instructional Media, Institute for Medical Education, University of Bern, Switzerland.
 
 
 
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