What are the goals of Polycythemia Vera treatment. How is PV managed in low-risk and high-risk patients. What medications and procedures are used to control hematocrit levels in PV. How can patients alleviate symptoms like itching in Polycythemia Vera.

Understanding Polycythemia Vera and Its Treatment Goals

Polycythemia Vera (PV) is a chronic myeloproliferative neoplasm characterized by an overproduction of red blood cells. While incurable, PV can be effectively managed for extended periods. The primary objectives of treatment are:

• Reducing thrombosis risk
• Alleviating symptoms
• Lowering the number of excess blood cells
• Maintaining hematocrit levels below 45% for men and 42% for women

What is the significance of hematocrit levels in PV management? Hematocrit, the percentage of red blood cells in total blood volume, serves as a crucial marker for disease control. Keeping hematocrit within normal ranges helps prevent complications and improves overall patient outcomes.

Risk Stratification in Polycythemia Vera

Effective treatment of PV begins with proper risk stratification. Patients are typically categorized into low-risk and high-risk groups based on specific criteria:

Low-Risk PV Patients

• Younger than 60 years old
• No history of thrombosis

High-Risk PV Patients

• 60 years or older
• History of thrombosis

Why is risk stratification important in PV treatment? Risk assessment helps tailor treatment approaches, ensuring that patients receive appropriate interventions based on their likelihood of developing complications, particularly thrombotic events.

Treatment Strategies for Low-Risk Polycythemia Vera Patients

Low-risk PV patients typically undergo less intensive management, focusing on preventive measures and symptom control. The treatment plan may include:

1. Regular monitoring for new clots or bleeding episodes
2. Management of cardiovascular risk factors
3. Low-dose aspirin therapy
4. Phlebotomy as needed

How does low-dose aspirin benefit PV patients? Aspirin therapy helps reduce the risk of blood clots, heart attacks, and strokes by preventing platelets from sticking together. This simple intervention can significantly impact patient outcomes, although it may cause side effects such as upset stomach and heartburn in some individuals.

Comprehensive Management for High-Risk Polycythemia Vera Patients

High-risk PV patients require a more intensive treatment approach to mitigate their increased risk of complications. In addition to the strategies employed for low-risk patients, high-risk individuals may receive:

• Cytoreductive medications to reduce blood cell counts
• More frequent monitoring and interventions

What are cytoreductive medications, and how do they work in PV treatment? Cytoreductive drugs are designed to decrease the production of blood cells, helping to control hematocrit levels and reduce the risk of thrombotic events. These medications may include hydroxyurea, ruxolitinib, interferon alfa, and busulfan, among others.

The Role of Therapeutic Phlebotomy in Polycythemia Vera Management

Therapeutic phlebotomy is a cornerstone of PV treatment for both low-risk and high-risk patients. This procedure involves:

• Regular blood draws to reduce blood cell counts
• Decreasing blood volume to improve circulation
• Alleviating symptoms such as headaches, itchiness, and vision problems

How does phlebotomy improve symptoms in PV patients? By reducing the number of red blood cells and overall blood volume, phlebotomy helps prevent blood “sludging” along vessel walls. This improvement in blood flow can lead to immediate relief of various PV-related symptoms, enhancing patient quality of life.

Medications for Cytoreduction in Polycythemia Vera

For high-risk PV patients, cytoreductive medications play a crucial role in disease management. Some commonly prescribed drugs include:

1. Hydroxyurea (Hydrea®): An oral chemotherapy agent that suppresses bone marrow production of blood cells
2. Ruxolitinib (Jakafi®): A JAK1/JAK2 inhibitor that helps reduce spleen size and alleviate PV symptoms
3. Interferon alfa: Available in various formulations, interferon helps regulate blood cell production
4. Busulfan (Myleran®): An alkylating agent used in cases where other treatments have been ineffective

What factors influence the choice of cytoreductive medication in PV treatment? The selection of a specific cytoreductive agent depends on various factors, including the patient’s age, disease severity, potential side effects, and individual response to treatment. Physicians often tailor medication choices to each patient’s unique circumstances.

Managing Pruritus: Strategies for Alleviating Itching in Polycythemia Vera

Itchy skin, or pruritus, is a common and often distressing symptom experienced by many PV patients. Several approaches can help manage this uncomfortable sensation:

• Modifying bathing habits (less frequent bathing, using cool water and gentle soaps)
• Avoiding hot tubs and hot showers
• Keeping skin well-moisturized
• Using antihistamines such as diphenhydramine or doxepin
• Light therapy (phototherapy) with psoralen and ultraviolet A (UVA) light
• Medications that block neurotransmitters involved in itching sensations (e.g., gabapentin, pregabalin)

Why is managing pruritus important in PV care? Chronic itching can significantly impact a patient’s quality of life and may lead to skin damage if left uncontrolled. Effective management of this symptom can greatly improve patient comfort and overall well-being.

The Importance of Clinical Trials in Advancing Polycythemia Vera Treatment

Clinical trials play a vital role in the development of new and more effective treatments for PV. Participation in these studies can offer several benefits:

• Access to cutting-edge therapies
• Contribution to medical advancements
• Potential for improved treatment outcomes
• Close monitoring by healthcare professionals

How can patients find and participate in PV clinical trials? The Leukemia & Lymphoma Society (LLS) offers one-on-one navigation services with Clinical Trial Specialists who can assist patients throughout the entire clinical trial process. This personalized support can help patients identify suitable trials and navigate the complexities of participation.

Types of Clinical Trials in Polycythemia Vera

Clinical trials for PV encompass a wide range of research objectives, including:

• Studies for newly diagnosed patients
• Trials focused on advanced disease management
• Research on treatments for patients intolerant or resistant to current medications

What makes clinical trials crucial for PV treatment advancement? By participating in clinical trials, patients not only gain access to potential new treatments but also contribute to the collective knowledge that drives progress in PV management. This collaborative effort between patients, researchers, and healthcare providers is essential for improving outcomes and quality of life for all individuals affected by PV.

Developing a Comprehensive Treatment Plan for Polycythemia Vera

Creating an effective treatment plan for PV requires careful consideration of various factors. Key elements in this process include:

1. Assessing the patient’s risk for thrombotic complications
2. Evaluating previous medical history, including any prior clotting events
3. Considering the patient’s age and overall health status
4. Discussing treatment goals and expected outcomes
5. Exploring all available treatment options, including clinical trials
6. Addressing potential side effects and management strategies

Why is individualized treatment planning crucial in PV management? Each patient’s medical situation is unique, and a one-size-fits-all approach is not effective in managing PV. Tailoring treatment plans to individual needs and risk factors helps optimize outcomes and minimize complications.

Enhancing Patient-Physician Communication

Effective communication between patients and their healthcare providers is essential for successful PV management. To facilitate this process:

• Prepare questions in advance of doctor’s visits
• Bring a support person to appointments for additional perspective and note-taking
• Consider recording discussions with the physician’s permission
• Seek clarification on any unclear information or medical terminology

How can improved patient-physician communication benefit PV treatment? Open and clear communication ensures that patients fully understand their condition, treatment options, and expected outcomes. This understanding empowers patients to actively participate in their care and make informed decisions about their PV management.

Long-Term Monitoring and Adjusting Treatment in Polycythemia Vera

PV is a chronic condition that requires ongoing management and monitoring. Long-term care typically involves:

• Regular blood tests to assess hematocrit levels and other blood parameters
• Periodic evaluations of treatment efficacy and side effects
• Adjustments to medication dosages or treatment strategies as needed
• Monitoring for disease progression or transformation
• Addressing new symptoms or complications as they arise

Why is long-term monitoring essential in PV management? Continuous assessment allows healthcare providers to track disease progression, evaluate treatment effectiveness, and make timely adjustments to ensure optimal control of PV. This proactive approach helps maintain quality of life and reduce the risk of complications over time.

The Role of Supportive Care in Polycythemia Vera

In addition to medical interventions, supportive care plays a crucial role in comprehensive PV management. This may include:

1. Nutritional counseling to support overall health
2. Physical activity recommendations to improve cardiovascular health
3. Psychological support to address the emotional impact of living with a chronic condition
4. Patient education programs to enhance understanding and self-management skills
5. Support groups or peer counseling to connect with others living with PV

How does supportive care contribute to better outcomes in PV patients? By addressing the physical, emotional, and social aspects of living with PV, supportive care measures can significantly improve patient well-being, treatment adherence, and overall quality of life.

Emerging Therapies and Future Directions in Polycythemia Vera Treatment

The landscape of PV treatment continues to evolve, with ongoing research into novel therapies and approaches. Some areas of active investigation include:

• New JAK inhibitors with improved efficacy and safety profiles
• Combination therapies targeting multiple pathways involved in PV pathogenesis
• Immunotherapies to modulate the immune response in PV
• Gene therapies aimed at correcting underlying genetic mutations
• Advanced monitoring techniques for early detection of disease progression or transformation

What potential benefits do emerging therapies offer for PV patients? As research progresses, new treatments may provide more effective disease control, reduced side effects, and improved quality of life for individuals living with PV. These advancements hold the promise of further enhancing the management of this chronic myeloproliferative neoplasm.

The Importance of Patient Advocacy and Education

As the field of PV treatment advances, patient advocacy and education play increasingly important roles. Key aspects include:

1. Raising awareness about PV and its impact on patients’ lives
2. Promoting access to optimal care and innovative treatments
3. Encouraging participation in clinical trials and research initiatives
4. Providing resources for patients to better understand and manage their condition
5. Fostering collaboration between patients, healthcare providers, and researchers

How does patient advocacy contribute to improving PV care? By empowering patients with knowledge and support, advocacy efforts can lead to better treatment outcomes, increased research funding, and improved overall care for the PV community.

https://www.lls.org/myeloproliferative-neoplasms/polycythemia-vera/treatment

Polycythemia Vera (PV) is a chronic disease: It’s not curable, but it can usually be managed effectively for very long periods. The goal of therapy is to reduce the risk of thrombosis and to ease symptoms by lowering the number of extra blood cells. 

Many treatment options are designed to manage PV by lowering hematocrit levels below 45 percent for men and 42 percent for women. Careful medical supervision and therapy is important to keep the hematocrit concentration at normal levels. 

Treatment for Low-Risk Patients

Patients are generally considered low risk if: 

• They are younger than 60 years, and 
• They have no history of thrombosis 

Treatment may include:

• Monitoring for new clots or bleeding
• Manage cardiovascular risk factors 
• Low-dose aspirin
• Phlebotomy

Treatment for High-Risk Patients 

Patients are generally considered high risk if: 

• They are 60 years or older, and/or 
• They have a history of thrombosis 

Treatment may include:

• Monitoring for new clots or bleeding
• Manage cardiovascular risk factors 
• Low-dose aspirin
• Phlebotomy
• Medication to reduce the number of blood cells (“cytoreductive” medication)

Low-Dose Aspirin

Low-dose aspirin may reduce the risk of blood clots, heart attacks and strokes. Low-dose aspirin helps prevent platelets from sticking together, making it less likely for blood clots to form. The most common side effects of aspirin are upset stomach and heartburn.

Therapeutic Phlebotomy

Most PV patients have their blood drawn regularly to reduce the number of blood cells and decrease blood volume. Phlebotomy is a procedure in which blood is taken from a vein similarly to what is done when donating blood. After phlebotomy, the blood is thinner and less likely to cause “sludging” (which occurs when red blood cells build up along walls of blood vessels). The immediate effect of phlebotomy is to decrease certain symptoms, such as headaches, itchiness, vision problems, ringing in the ears and dizziness.

Medications to Reduce the Number of Blood Cells

High-risk PV patients may be prescribed cytoreductive drugs to reduce the number of blood cells. These drugs may include any or a combination of the drugs listed below

• Hydroxyurea (Hydrea®)
• Ruxolitinib (Jakafi®)
• Interferon alfa (Intron® A, BESREMi® [alfa-2b], and Roferon®-A [alfa-2a]), and sustained-release preparations of these called PEG-Intron® ([peginterferon alfa-2b] and Pegasys® [peginterferon alfa-2a])
• Busulfan (Myleran®)

Treatments to Reduce Itching

A troublesome symptom that occurs in many PV patients is itchy skin (“pruritus”). Treatment options include:

• Bathe less frequently.
• Bathe or shower in cool water and use a gentle soap.
• Avoid hot tubs, heated whirlpools and hot showers or baths. 
• Keep skin well moisturized with lotion and try not to scratch it because that can damage the skin.
• Antihistamines such as diphenhydramine (Benadryl®) or doxepin may help itching that does not go away.
• Light therapy (phototherapy) using a medicine called “psoralen” combined with ultraviolet A (UVA) light.
• Medications such as gabapentin or pregabalin that block neurotransmitters in the central nervous system from sending signals that trigger itching.

For information about the drugs listed on this page, visit Drug Listings.

Clinical Trials

Taking part in a clinical trial may be the best treatment choice for some  PV patients. Patient participation in clinical trials is important in the development of new and more effective treatments for PV and may provide patients with additional treatment options.  

Click here to read more about clinical trials.

Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

There are clinical trials for newly diagnosed patients, for patients with advanced disease, and for patients who are intolerant of or resistant to their current medications.

Treatment Planning

Treatment decisions are based on the patient’s risk for clotting complications (thrombosis). The two main risk factors for thrombosis are:

•  A previous clot or clots
• Age 60 years or older

Every patient’s medical situation is different and should be evaluated individually by a hematologist-oncologist. As you develop a treatment plan with your doctor,  it is important to discuss:

• The results you can expect from treatment
• Potential side effects
• All treatment options, including treatments being studied in clinical trials

You may find it helpful to bring a loved one with you to your doctor’s visits for support and to take notes and ask follow-up questions. It’s a good idea to prepare questions you’d like to ask when you visit your doctor. You can also record your conversations with your doctor and listen more closely when you get home.

To download lists of suggested questions to ask your healthcare providers, click here.

Related Links

• Download or order The Leukemia & Lymphoma Society’s free booklet Myeloproliferative Neoplasms.

Approach Considerations, Medical Care, Phlebotomy

1. National Cancer Institute. PDQ® Chronic Myeloproliferative Neoplasms Treatment. National Cancer Institute. Available at http://www.cancer.gov/types/myeloproliferative/hp/chronic-treatment-pdq#link/_5. November 4, 2020; Accessed: September 16, 2021.

2. Tefferi A, Vannucchi AM, Barbui T. Polycythemia vera: historical oversights, diagnostic details, and therapeutic views. Leukemia. 2021 Sep 3. 12(4):339-51. [QxMD MEDLINE Link].

3. Lu X, Chang R. Polycythemia Vera. 2021 Jan. 5(5):327-31. [QxMD MEDLINE Link]. [Full Text].

4. Streiff MB, Smith B, Spivak JL. The diagnosis and management of polycythemia vera in the era since the Polycythemia Vera Study Group: a survey of American Society of Hematology members’ practice patterns. Blood. 2002 Feb 15. 99(4):1144-9. [QxMD MEDLINE Link]. [Full Text].

5. Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016 May 19. 127 (20):2391-405. [QxMD MEDLINE Link]. [Full Text].

6. James C, Ugo V, Le Couedic JP, et al. A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature. 2005 Apr 28. 434(7037):1144-8. [QxMD MEDLINE Link].

7. Kralovics R, Teo SS, Buser AS, et al. Altered gene expression in myeloproliferative disorders correlates with activation of signaling by the V617F mutation of Jak2. Blood. 2005 Nov 15. 106(10):3374-6. [QxMD MEDLINE Link]. [Full Text].

8. Levine RL, Wadleigh M, Cools J, et al. Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell. 2005 Apr. 7(4):387-97. [QxMD MEDLINE Link]. [Full Text].

9. Guglielmelli P, Barosi G, Pieri L, et al. JAK2V617F mutational status and allele burden have little influence on clinical phenotype and prognosis in patients with post-polycythemia vera and post-essential thrombocythemia myelofibrosis. Haematologica. 2009 Jan. 94(1):144-6. [QxMD MEDLINE Link]. [Full Text].

10. Mustjoki S, Borze I, Lasho TL, et al. JAK2V617F mutation and spontaneous megakaryocytic or erythroid colony formation in patients with essential thrombocythaemia (ET) or polycythaemia vera (PV). Leuk Res. 2009 Jan. 33(1):54-9. [QxMD MEDLINE Link].

11. Vannucchi AM. From leeches to personalized medicine: evolving concepts in the management of polycythemia vera. Haematologica. 2017 Jan. 102 (1):18-29. [QxMD MEDLINE Link]. [Full Text].

12. Palandri F, Mora B, Gangat N, Catani L. Is there a gender effect in polycythemia vera?. Ann Hematol. 2021 Jan. 100 (1):11-25. [QxMD MEDLINE Link]. [Full Text].

13. Rusak T, Ciborowski M, Uchimiak-Owieczko A, Piszcz J, Radziwon P, Tomasiak M. Evaluation of hemostatic balance in blood from patients with polycythemia vera by means of thromboelastography: The effect of isovolemic erythrocytapheresis. Platelets. 2011 Nov 18. [QxMD MEDLINE Link].

14. Spivak JL, Considine M, Williams DM, Talbot CC Jr, Rogers O, Moliterno AR, et al. Two clinical phenotypes in polycythemia vera. N Engl J Med. 2014 Aug 28. 371(9):808-17. [QxMD MEDLINE Link].

15. Cabagnols X, Favale F, Pasquier F, Messaoudi K, Defour JP, Ianotto JC, et al. Presence of atypical thrombopoietin receptor (MPL) mutations in triple-negative essential thrombocythemia patients. Blood. 2016 Jan 21. 127 (3):333-42. [QxMD MEDLINE Link].

16. Tefferi A, Barbui T. Essential Thrombocythemia and Polycythemia Vera: Focus on Clinical Practice. Mayo Clin Proc. 2015 Sep. 90 (9):1283-93. [QxMD MEDLINE Link].

17. Barbui T, Thiele J, Gisslinger H, Finazzi G, Carobbio A, Rumi E, et al. Masked polycythemia vera (mPV): results of an international study. Am J Hematol. 2014 Jan. 89 (1):52-4. [QxMD MEDLINE Link]. [Full Text].

18. Wang JC, Shi G, Baptiste S, Yarotska M, Sindhu H, Wong C, et al. Quantification of IGF-1 Receptor May Be Useful in Diagnosing Polycythemia Vera-Suggestion to Be Added to Be One of the Minor Criterion. PLoS One. 2016 Nov 3. 11 (11):e0165299. [QxMD MEDLINE Link]. [Full Text].

19. Bose P, Verstovsek S. JAK2 inhibitors for myeloproliferative neoplasms: what is next?. Blood. 2017 Jul 13. 130 (2):115-125. [QxMD MEDLINE Link].

20. McMullin MF, Wilkins BS, Harrison CN. Management of polycythaemia vera: a critical review of current data. Br J Haematol. 2015 Oct 22. [QxMD MEDLINE Link].

21. FDA Approves Treatment for Rare Blood Disease. U.S. Food & Drug Administration. Available at https://www.fda.gov/news-events/press-announcements/fda-approves-treatment-rare-blood-disease. November 12, 2021; Accessed: November 20, 2021.

22. Squizzato A, Romualdi E, Passamonti F, Middeldorp S. Antiplatelet drugs for polycythaemia vera and essential thrombocythaemia. Cochrane Database Syst Rev. 2013 Apr 30. 4:CD006503. [QxMD MEDLINE Link].

23. Alvarez-Larrán A, Martínez-Avilés L, Hernández-Boluda JC, Ferrer-Marín F, Antelo ML, Burgaleta C, et al. Busulfan in patients with polycythemia vera or essential thrombocythemia refractory or intolerant to hydroxyurea. Ann Hematol. 2014 Jul 2. [QxMD MEDLINE Link].

24. Alvarez-Larrán A, Kerguelen A, Hernández-Boluda JC, et al. Frequency and prognostic value of resistance/intolerance to hydroxycarbamide in 890 patients with polycythaemia vera. Br J Haematol. 2016 Mar. 172 (5):786-93. [QxMD MEDLINE Link].

25. Barbui T, Masciulli A, Marfisi MR, Tognoni G, Finazzi G, Rambaldi A, et al. White blood cell counts and thrombosis in polycythemia vera: a subanalysis of the CYTO-PV study. Blood. 2015 Jul 23. 126 (4):560-1. [QxMD MEDLINE Link]. [Full Text].

26. Bewersdorf JP, Giri S, Wang R, Podoltsev N, Williams RT, Tallman MS, et al. Interferon alpha therapy in essential thrombocythemia and polycythemia vera-a systematic review and meta-analysis. Leukemia. 2021 Jun. 35 (6):1643-1660. [QxMD MEDLINE Link]. [Full Text].

27. Abu-Zeinah G, Krichevsky S, Cruz T, Hoberman G, Jaber D, Savage N, et al. Interferon-alpha for treating polycythemia vera yields improved myelofibrosis-free and overall survival. Leukemia. 2021 Sep. 35 (9):2592-2601. [QxMD MEDLINE Link].

28. webmd.com”>Gisslinger H, Klade C, Georgiev P, Krochmalczyk D, Gercheva-Kyuchukova L, Egyed M, et al. Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study. Lancet Haematol. 2020 Mar. 7 (3):e196-e208. [QxMD MEDLINE Link].

29. Besremi. European Medicines Agency. Available at https://www.ema.europa.eu/en/medicines/human/EPAR/besremi. February 22, 2021; Accessed: September 20, 2021.

30. Marchioli R, et al; CYTO-PV Collaborative Group. Cardiovascular events and intensity of treatment in polycythemia vera. N Engl J Med. 2013 Jan 3. 368 (1):22-33. [QxMD MEDLINE Link].

31. Berk PD, Goldberg JD, Donovan PB, et al. Therapeutic recommendations in polycythemia vera based on Polycythemia Vera Study Group protocols. Semin Hematol. 1986 Apr. 23(2):132-43. [QxMD MEDLINE Link].

32. Weinfeld A, Swolin B, Westin J. Acute leukaemia after hydroxyurea therapy in polycythaemia vera and allied disorders: prospective study of efficacy and leukaemogenicity with therapeutic implications. Eur J Haematol. 1994 Mar. 52(3):134-9. [QxMD MEDLINE Link].

33. Fruchtman SM, Mack K, Kaplan ME, et al. From efficacy to safety: a Polycythemia Vera Study Group report on hydroxyurea in patients with polycythemia vera. Semin Hematol. 1997 Jan. 34(1):17-23. [QxMD MEDLINE Link].

34. Huang BT, Zeng QC, Zhao WH, Li BS, Chen RL. Interferon a-2b gains high sustained response therapy for advanced essential thrombocythemia and polycythemia vera with JAK2V617F positive mutation. Leuk Res. 2014 Jul 15. [QxMD MEDLINE Link].

35. webmd.com”>Landolfi R, Marchioli R, Kutti J, et al. Efficacy and safety of low-dose aspirin in polycythemia vera. N Engl J Med. 2004 Jan 8. 350(2):114-24. [QxMD MEDLINE Link]. [Full Text].

36. Vannucchi AM. Ruxolitinib versus standard therapy for the treatment of polycythemia vera. N Engl J Med. 2015 Apr 23. 372 (17):1670-1. [QxMD MEDLINE Link]. [Full Text].

37. Pardanani A, Harrison C, Cortes JE, Cervantes F, Mesa RA, Milligan D, et al. Safety and Efficacy of Fedratinib in Patients With Primary or Secondary Myelofibrosis: A Randomized Clinical Trial. JAMA Oncol. 2015 Aug. 1 (5):643-51. [QxMD MEDLINE Link].

38. Slakey DP, Klein AS, Venbrux AC, Cameron JL. Budd-Chiari syndrome: current management options. Ann Surg. 2001 Apr. 233(4):522-7. [QxMD MEDLINE Link]. [Full Text].

39. webmd.com”>Tefferi A, Barbui T. Polycythemia vera and essential thrombocythemia: 2017 update on diagnosis, risk-stratification, and management. Am J Hematol. 2017 Jan. 92 (1):94-108. [QxMD MEDLINE Link].

40. Khanal N, Giri S, Upadhyay S, Shostrom VK, Pathak R, Bhatt VR. Risk of second primary malignancies and survival of adult patients with polycythemia vera: A United States population-based retrospective study. Leuk Lymphoma. 2016. 57 (1):129-33. [QxMD MEDLINE Link].

41. Nelson R. FDA Approves Fedratinib for the Treatment of Myelofibrosis. Medscape Medical News. Available at https://www.medscape.com/viewarticle/916928. August 16, 2019; Accessed: August 16, 2019.

42. Abdulkarim K, Girodon F, Johansson P, et al. AML transformation in 56 patients with Ph- MPD in two well defined populations. Eur J Haematol. 2009 Feb. 82(2):106-11. [QxMD MEDLINE Link].

43. Siebolts U, Breuhahn K, Hennecke A, Schultze JL, Wickenhauser C. Imbalance of DNA-dependent protein kinase subunits in polycythemia vera peripheral blood stem cells. Int J Cancer. 2009 Feb 1. 124(3):600-7. [QxMD MEDLINE Link].

44. Gotlib J, Kiladjian JJ, Vannucchi A, Rambaldi A, Reiter A, Shomali W, et al. A Phase 2 Study of Pemigatinib (FIGHT-203; INCB054828) in Patients with Myeloid/Lymphoid Neoplasms (MLNs) with Fibroblast Growth Factor Receptor 1 (FGFR1) Rearrangement (MLN FGFR1). Blood. 2021 Nov 23(138(suppl 1): 385. Presented at the 64th American Society of Hematology Virtual Meeting 2021. [Full Text].

Polycythemia in newborns. What is polycythemia in newborns?

IMPORTANT
The information in this section should not be used for self-diagnosis or self-treatment. In case of pain or other exacerbation of the disease, only the attending physician should prescribe diagnostic tests. For diagnosis and proper treatment, you should contact your doctor.

Polycythemia in newborns is a syndrome of increased concentration of cellular elements in the blood (mostly erythrocytes). In the clinic, there is depression of the central nervous system and signs of plethora: cherry cyanosis, increased respiration and heart rate, etc. Blood thickening is manifested by microcirculation disorders, leading to multiple organ failure with the possible development of heart attacks in various organs. The diagnosis is confirmed by laboratory testing with a central venous hematocrit greater than 65%. Treatment of polycythemia in newborns is partial exchange blood transfusion. The underlying disease is also treated.

• Causes of polycythemia in newborns
• Classification and symptoms of polycythemia in newborns
• Diagnosis of polycythemia in newborns
• Treatment of polycythemia in newborns
  • Prognosis and prevention of polycythemia in newborns
• Prices for treatment

General

Polycythemia in newborns is associated with an increase in the total number of blood cells of all three germs, so erythrocytosis should not be confused with this syndrome, when only the number of red blood cells increases. However, in this case, the erythrocyte germ is subject to enhanced growth to a greater extent. The syndrome occurs in 0.4-12% of newborns, it is more common in premature babies, however, late births also increase the risk of developing polycythemia in newborns, since dehydration occurs during postmaturity, leading to blood clotting. The relevance of the syndrome in pediatrics is associated with the long-term consequences of hypoxia, especially for the brain, which always entails neurological consequences to one degree or another. The child may lag behind in development, suffer from violations of social adaptation, etc.

Polycythemia in newborns

Causes of polycythemia in newborns

An increase in the number of blood cells, especially erythrocytes, is associated with insufficient oxygen supply, while hypoxia can develop both in utero and after birth. In the first case, placental pathologies become the cause, since it is through the placental vessels that the fetus receives oxygen. This may be fetoplacental insufficiency, anomalies of the vessels of the placenta, tuberculosis, etc. A certain role is played by the bad habits of the mother, in particular, smoking, which also contributes to intrauterine hypoxia. Insufficient supply of oxygen is also observed in the presence of heart defects in the mother. Occasionally, late ligation of the umbilical cord is involved in the pathogenesis, which leads to significant hypervolemia in the first hours of the baby’s life.

Polycythemia in newborns is also associated with many causes on the part of the child. The development of this condition is caused by malformations of the cardiovascular system and the pulmonary apparatus, intrauterine infections, diseases of the adrenal glands and the thyroid gland. Some genetic defects can also be manifested by an increase in the number of blood cells. Such a violation occurs with chromosomal abnormalities – Down’s disease, Beckwith-Wiedemann syndrome, trisomy 13, etc. Hemoglobinopathies, in which red blood cells are more difficult to part with an oxygen molecule, also lead to hypoxia and, as a result, to polycythemia in newborns.

The pathogenesis of this condition is due to the high viscosity of the blood, which is uncharacteristic for a child of this age. As a result, all internal organs begin to suffer from increased stress. This is of particular importance in the case of vital organs (heart, lungs and brain), however, disturbances in the activity of other organs and systems can also contribute to the worsening of the child’s condition and worsening of the prognosis. Most of the clinical symptoms of polycythemia in newborns actually reflect the consequences of microcirculation disorders associated with multiple blockage of capillaries by erythrocyte sludge (clusters of cells).

Classification and symptoms of polycythemia in newborns

Allocate primary and secondary polycythemia in newborns. Primary (true) polycythemia is associated with damage to the hematopoietic germ, due to which there is an inadequately high increase in the number of erythrocytes, leukocytes and platelets. Benign familial polycythemia also refers to the primary forms of pathology. All other options are a reaction to changes in the environment (for example, hypoxia) or in internal organs (malformations, etc.), and therefore are considered secondary. Polycythemia in newborns is divided into normovolemic and hypervolemic, with the latter not only the number of blood cells is increased, but also the volume of its liquid fraction.

The main symptom of polycythemia in newborns, indicating plethora, is plethora. It is a characteristic cherry skin tone, combined with peripheral cyanosis. The child is outwardly lethargic, sucks poorly, there is hypotension, muscular dystonia, tremor, convulsions, and apnea attacks are possible. Less common is increased nervous excitability. Breathing and heart rate increase as the body tries to cope with increased blood volume and increased blood viscosity. There are phenomena of multiple organ failure, manifested by reduced cardiac output, pulmonary hypertension, and respiratory distress syndrome.

There may be signs of intraventricular hemorrhage and cerebral infarction. On the part of the gastrointestinal tract, symptoms such as regurgitation and vomiting are noted, sometimes necrotizing enterocolitis of newborns develops and even spontaneous perforation of the intestinal wall. Often, a clinic of acute renal failure joins, which is manifested by the presence of protein or blood in the urine, dysuric phenomena, etc. Renal vein thrombosis and priapism are possible. As can be seen from the above list of symptoms, the clinic of polycythemia in newborns is diverse and non-specific, which greatly complicates the timely establishment of an accurate diagnosis. In about 40% of cases, symptoms are mild or absent.

Diagnosis of polycythemia in newborns

Polycythemia in newborns does not have any pathognomonic manifestations. A plethora allows a pediatrician to suspect a pathology during a physical examination. In general, the diagnosis is based on the results of laboratory tests. An important indicator is the central venous hematocrit, which in this condition exceeds 65%. Biochemical blood tests always detect hypoglycemia, hypocalcemia, hypomagnesemia. The remaining diagnostic measures are aimed at identifying the cause of polycythemia in newborns.

Heart defects are confirmed by ECG and echocardiography. Anomalies of development and diseases of the lungs are determined by X-ray examination. If each specific nosology is suspected, its own diagnostic methods are used. It is important to understand that polycythemia in newborns may be a variant of the norm. It is also important to distinguish this condition from blood clotting, when polycythemia is relative and occurs due to a decrease in the volume of the liquid part of the blood. This happens with dehydration, for example, with prolonged phototherapy or being under a source of radiant heat, problems with enteral nutrition (frequent regurgitation, loose stools, including those of infectious origin), etc.

Treatment of polycythemia in newborns

The tactics of therapy is determined by two components: central venous hematocrit and the presence or absence of clinical manifestations. Often the indicators of central venous hematocrit correspond to polycythemia in newborns, and the child’s condition remains good, there are no signs of microcirculation disorders. In this case, expectant management is recommended with constant monitoring of hematocrit and the state of internal organs. The exception is when the venous hematocrit exceeds 70%. This is an indication for the start of therapeutic measures even without symptoms.

If polycythemia in newborns is clinically manifest, partial exchange transfusion becomes the only treatment. According to a specially derived formula, the volume of blood that is taken from the child is determined. Instead, a saline transfusion is performed. In this way, hemodilution is achieved, that is, the restoration of the normal concentration of cellular elements in the blood, which leads to the elimination of microcirculatory disorders. Protein solutions are not used, since they can cause an increase in the concentration of fibrinogen, which is also atypical for the blood composition of a newborn, and therefore represents an additional danger.

Prognosis and prevention of polycythemia in newborns

The prognosis is determined by the underlying disease, but, as a rule, remains unfavorable. In most cases, hypoxia becomes the cause of polycythemia in newborns, and it is detrimental to the brain, as it leads to irreversible destructive changes. In the future, such children may lag behind in development (ZPR, ZRR, mental retardation), disability is possible. Of particular danger are asymptomatic cases, which may go unnoticed for a long time. Prevention of polycythemia in newborns is possible at the prenatal stage and consists in eliminating the possible causes of hypoxia. Fetoplacental insufficiency is treated and the somatic condition of the mother is corrected, a pregnant woman is recommended to give up bad habits, etc.

You can share your medical history, what helped you in the treatment of polycythemia in newborns.

Sources

1. self-treatment. In case of pain or other exacerbation of the disease, only the attending physician should prescribe diagnostic tests. For diagnosis and proper treatment, you should contact your doctor.

   CBC summary in Israel : Armedical

   Treatment in Israel
   State
   hospital
   Ichilov-Surasky. Official representative.

   CBC (Complete Blood Count) – complete blood count. A complete blood count is an important tool in the diagnosis of diseases of the general functioning of the human body. At the Tel Aviv Ichilov Hospital, a complete blood count is performed in a computerized laboratory environment. A complete blood count (CBC) provides quantitative and qualitative information about the conditions for a decrease in red blood cells, which indicates anemia, infection, and various disorders.

   In the laboratory of the Ichilov hospital, in most cases, a blood sample is taken from a vein, in some cases from a finger, this is the case when it is necessary to obtain the results of a blood test in a short period of time.

   Red blood cells (RBC) are red blood cells that carry hemoglobin. The main function of erythrocytes is to transport oxygen from the lungs to all tissues and carbon dioxide from the tissues back to the lungs. Few erythrocytes – little hemoglobin. Too little hemoglobin means too few red blood cells. They are interconnected.

   Normal erythrocytes : 4.2 to 5.6 (number of cells per microliter of blood)

   Lack of red blood cells is one of the signs of anemia (anemia). Anemia can be caused by iron deficiency. An excess of red blood cells is called polycythemia. Polycythemia can be caused by a defect in the production of red blood cells in the bone marrow.

   Hemoglobin (HGB) – A protein found in red blood cells that is responsible for transporting oxygen molecules to the cells of the body. Hemoglobin is an iron-containing complex that surrounds protein. The level of hemoglobin is not a constant value and depends on age, gender, ethnicity, disease, smoking, in women – on pregnancy, etc.

   Normal hemoglobin : 11.7 – 17.0 gr.dl. (g per 100 ml of blood). There are options depending on gender and age.

   Reduced hemoglobin level – severe anemia. Anemia can be caused by a significant lack of red blood cells, acute or chronic blood loss. This is due to a lack of folic acid B9 and vitamin B12. Hemolysis (increased destruction of the membranes of red blood cells) from autoimmune conditions, infections and hereditary diseases of blood formation.

   Mean volume of hemoglobin (MCH) is the average amount of hemoglobin in a single erythrocyte: in red blood cells.

   Norm : 27-31pg.

   Exception: Reduced amount of hemoglobin in red blood cells. Anemia and other hematological problems.

   Hematocrit (HCT) – shows as a percentage the index of the volume of erythrocytes to the volume of the whole blood sample.

   Norm (HCT): men 42% -52%, women – from 36% to 48%

   Exception: A low hematocrit indicates anemia (polycythemia). Elevated hematocrit – erythremia.

   Mean erythrocyte volume (MCV) is the index of the mean erythrocyte volume.

   Norm (MCV): 80-94. Values ​​depend on age.

   Exception: An abnormally low reading is called microcytosis. This may be due to iron deficiency, iron absorption in the intestines, kidney disease, thalassemia, genetic causes, and blood loss. An abnormally high reading is called macrocytosis.
   Lack of vitamins B9 and B12, disruption of the thyroid gland.

   Red blood cell distribution width in percent (RDW) – red blood cell size difference.

   Normal (RDW): 9%- 14.5%

   Exception: Abnormal levels may indicate impaired hematopoiesis.

   Leukocytes (WBC) are white or colorless blood cells of various sizes. The main function of leukocytes is to counteract infections, viruses, bacteria, etc. Leukocytes are divided into 5 types: neutrophils, lymphocytes, monocytes, eosinophils and basophils.

   Normal (WBC): 4.50 to 11.0 (from 4,500 to 11,000 cubic cells per mC of blood)
   Exception: a significant increase or a significant decrease in leukocytes indicates an inflammatory process. The type of leukocytes indicates the nature of the infection.

   Neutrophils (NEUT) – Percentage of neutrophils in the blood. Neutrophils are generated in the bone marrow. Their service life in the blood lasts several hours. Neutrophils destroy microbes (phagocytosis).

   Norm (NEUT): 40%-75% (neutrophil count in 100 leukocytes). Normal values ​​depend on age.

   Exception: Elevated neutrophil count indicates inflammation and infection.

   Lymphocytes (LYM) are the main cells of the human immune system. Lymphocytes are a type of white blood cell that is produced in the lymphatic system and bone marrow. According to their functions, lymphocytes are divided into B-lymphocytes, which produce antibodies, T-lymphocytes, which fight infections, and NK lymphocytes, which control the quality of body cells.

   Norm (LYMLYMPH): from 20% to 40% (number of lymphocytes in 100 leukocytes). The normal value varies with age (especially in children).

   Exception: An increased number of lymphocytes can be caused by various diseases, especially viral infections.

   Eosinophils (EOSEOSIN) are white blood cells characterized by a specific orange color. They take part in the immune system. Elevated in infections with parasites. There is a tendency to appear in allergies and asthma.

   Normal (EOS): 0% to 6% (eosinophils per 100 leukocytes)

   Exception: An increase in the percentage of eosinophils is a sign of the presence of allergy factors or helminthic invasions.

   Monocytes (MONO) is one of the types of phagocytes, the largest type of leukocytes. Monocytes are formed in the bone marrow. These cells are involved in the regulation and differentiation of hematopoiesis, then go to the tissues of the body and there they turn into macrophages. Monocytes are of great importance, as they are responsible for the initial activation of the entire human immune system.

   Normal (MONO): 3% to 7% (of total white blood cells)

   Exception: An increase in the percentage of monocytes is a sign of the development of various viral diseases.

   Basophils (BASO) is one of the major forms of white blood cells associated with the immune system. The main function is the expansion of blood vessels during infection.

   Normal (BASO): 0% to 2% (of total white blood cells)

   Exception: An increase in the percentage of basophil cells is a sign of infection or various viral infections.

   Platelets (PLT) – Cells that affect blood coagulation. Platelets are responsible for hemostasis, wound healing and bleeding control.