TREATMENT STRATEGIES FOR POLYCYTHEMIA VERA: OBSERVATIONS IN A DUTCH ‘REAL-WORLD’ COHORT STUDY
Author(s): ,
Claire van de Ree- Pellikaan
Affiliations:
Internal Medicine,Albert Schweitzer Hospital,Dordrecht,Netherlands
,
Arne de Kreuk
Affiliations:
Internal Medicine,OLVG,Amsterdam,Netherlands
,
Cees Schaar
Affiliations:
Internal Medicine,Gelre Hospital,Apeldoorn,Netherlands
,
Aart Beeker
Affiliations:
Internal Medicine,Spaarne Gasthuis,Hoofddorp,Netherlands
,
Ellen Dompeling
Affiliations:
Internal Medicine,Isala Klinieken,Zwolle,Netherlands
,
Cees Gerrits
Affiliations:
Internal Medicine,Ziekenhuisgroep Twente,Hengelo,Netherlands
,
Anja van Houten
Affiliations:
Internal Medicine,Maasstad Hospital,Rotterdam,Netherlands
,
Martin Schipperus
Affiliations:
Hematology,Haga Teaching Hospital,The Hague,Netherlands
,
Leonie Strobbe
Affiliations:
Internal Medicine,Gelre Hospital,Zutphen,Netherlands
,
Ward Posthuma
Affiliations:
Internal Medicine,Reinier de Graaf Hospital,Delft,Netherlands
,
Karin Klauke
Affiliations:
Novartis Pharma B.V.,Arnhem,Netherlands
Peter Westerweel
Affiliations:
Internal Medicine,Albert Schweitzer Hospital,Dordrecht,Netherlands
EHA Library. Westerweel P. 06/16/18; 215670; PS1370
Dr. Peter Westerweel
Dr. Peter Westerweel
Contributions
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Abstract

Abstract: PS1370

Type: Poster Presentation

Presentation during EHA23: On Saturday, June 16, 2018 from 17:30 - 19:00

Location: Poster area

Background

Major thrombotic events are the main cause of mortality in Polycythemia Vera (PV) and therefore the primary focus of therapy. Treatment of PV requires control of circulating erythrocyte volume in addition to antiplatelet and/or anticoagulant treatment. Reduction of erythrocyte volume may be achieved by phlebotomy/erythrocytaferesis (Phleb), hydroxyurea treatment (HU) or a combination of both (Phleb+HU). Other treatments such as JAK2 inhibition and interferon are used in selected cases. In the absence of data from clinical trials comparing treatment strategies, there is a large variation in clinical practice.

Aims

Assessment of ‘real-world’ clinical practice of choice of treatment strategies for cytoreduction in PV patients and effects on the course of haematological control and patient outcome.

Methods

A retrospective chart review was performed in 150 PV patients from 10 major non-academic hospitals in the Netherlands to reflect a ‘real-world’ population of non-referred patients. Patients were required to meet the WHO 2008 diagnostic criteria. All patients provided written informed consent. Data were retrospectively collected from the time of diagnosis to inclusion based on the hospital records by trained data managers.

Results

Patients had a median age of 64 (range 30-91) at diagnosis and 49% was male. 96.4% was JAK2 mutation positive. There was a high prevalence of a history of prior vascular events (37%) and concomitant cardiovascular risk factors (hypertension 59%, hypercholesterolemia 20%, diabetes 12%). A majority of patients received a combination of Phleb+HU (n=60, 40%), while others received HU only (n=32, 21%), only Phleb (n=37, 35%) or other treatment combinations (n=21, 14%). Table 1 shows the haematological parameters at baseline and at 6 and 12 months for patients treated with Phleb, HU or Phleb+HU. There were no statistically significant differences. The average (±SD) time to achieve a hematocrit target of 0.45 was the shortest in Phleb only patients (148±225 days) compared to HU (490±620 days) and Phleb+HU (346±426 days) treated patients. Leukocyte and platelet levels were numerically lower in HU-treated patients and ELN response targets were more often reached (P<0.05 in ANOVA). During the median follow-up period of 4.1 years after PV diagnosis, 22 patients (15%) suffered from a thrombotic vascular event despite treatment. In patients experiencing an event, the distribution between treatment strategies was similar and 59% used HU, while this was 61% in patients that remained event-free.

Conclusion

We observed major clinical variation in treatment strategies for PV in Dutch clinical practice. Although phlebotomizing patients shortens the time to achieving hematocrit control, median hematocrit levels were very similar after 6-12 months of treatment to that with HU alone or in combination with phlebotomies. In contrast, achievement of platelet and leukocyte ELN responses was higher in HU treated patients. Interestingly, the median hematocrit values achieved in Dutch clinical practice (43.0-44.0%) are comparable to the low-hematocrit arm of the CYTO-PV trial (44.4%). Nonetheless, the thrombotic vascular event rate in our cohort of Dutch PV patients remained clinically significant.

Session topic: 16. Myeloproliferative neoplasms - Clinical

Keyword(s): Polycythemia vera

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