Guidelines Utility of guidelines Standardise treatment Education Better use of health resource Reimbursement Endpoints for clinical trials 2 Investigation and management of erythrocytosis ID: 920322
Download Presentation The PPT/PDF document "State of play across the globe: no stand..." is the property of its rightful owner. Permission is granted to download and print the materials on this web site for personal, non-commercial use only, and to display it on your personal computer provided you do not modify the materials and that you retain all copyright notices contained in the materials. By downloading content from our website, you accept the terms of this agreement.
Slide1
State of play across the globe: no standard guidelines for MPN
Slide2Guidelines:
Utility of guidelines…
Standardise treatment
EducationBetter use of health resourceReimbursement (?)Endpoints for clinical trials
2
Slide3Investigation and management of
erythrocytosis
HERE THRESHOLD FOR HIGH RISK PV = 65 years
Ditto for thrombocytosis
Management of Myelofibrosis
Investigation and management of eosinophilia
Lab diagnostic approaches to MPN
Slide4ELN first 2011 revised 2018
New criteria for diagnosis
Lower
Hb threshold for PVAddition of newer genomicsEquivalence of IFN to HUDissonance with NCCN and ESMO re ruxo in MF
Slide5ESMO guidelines for MF
5
*Dynamic
IPSS and Dynamic IPSS-plus after diagnosis.
**Hydroxyurea for symptomatic splenomegaly in countries
where ruxolitinib is not approved for low-risk patients.
If anaemia is the problem, erythropoietin, corticosteroids, danazol,
immunomodulators or splenectomy. ***
For patients presenting with
symptomatic splenomegaly
and/or constitutional symptoms if allowed by the label.
§
For patients presenting with symptomatic splenomegaly and/or constitutional symptoms.
Slide6NCCN Guidelines:
Myeloproliferative Neoplasms (MPNs) Inaugural 2016
Treatment Guidelines
Myelofibrosis
Treatment Guidelines
(Forthcoming 2017)
Polycythemia Vera
Essential Thrombocythemia
Diagnosis & Treatment
(Forthcoming 2017)
Atypical MPNs
HES, SMCD, etc.
Diagnosis &
Treatment
Response
Myelofibrosis
Polycythemia Vera
Essential
Thrombocythemia
Disease Burden & Treatment Planning
Myelofibrosis
Polycythemia Vera
Essential
Thrombocythemia
Slide7Evolving Management of MPNs
Workup of an MPN
Diagnosis, risk stratification, symptom burden, and treatment goals
NCCN Treatment Guidelines - Myelofibrosis
Low & intermediate 1 riskIntermediate 2 & high riskProgressive MF
Supportive care and management of MF-associated Anemia
Slide8MPN Workup
Diagnosis
Endorsement of WHO 2016
Disease BurdenMPN SAF / MPN10PrognosisIPSS at Dx; DIPSS subsequent. New Molecular Features
Slide9Evolving Management of MPNs
Workup of an MPN
Diagnosis, risk stratification, symptom burden, and treatment goals
NCCN Treatment Guidelines - Myelofibrosis
Low & intermediate 1 riskIntermediate 2 & high riskProgressive MF
Supportive care and management of MF-associated Anemia
Slide10Slide11Slide12Slide13Slide14Slide15Slide16So …… a clinical case
45 years old
ffemale
diagnosed with ETCALR positiveWbc 9Hb 13Platelets 1245What treatment?
Slide17According to current guidelines
BCSH, NCCN, ESMO
and ELN
Aspirin onlyCZEMPAspirin plus anagrelide or IFN
Slide18However…
Slide19ET
Retrospective study of low-risk ET patients on
antiplatelets
only or observationCALR mutant (n=271): start cytoreduction earlier, most often due to high plts no difference in thrombosis with
antiplatelets higher incidence of bleeding with antiplateletsJAK2V617F (n=162): reduced incidence of venous
thrombosis with antiplatelets no effect on bleeding risk
B
leeding
P=0.03
Venous thrombosis
P=0.045
Slide20This data is not reflected in ANY current guidelines
…
Other aspects that are currently missing
Management of JAK2 pos ET when the Hb goes upSwitching therapy – will be more important with JAK2i in MFHow to manage children with MPN
And other areas
Slide21Disadvantages of guidelines
May not be up to date
May encourage formulaic practice
May not reflect locally available therapy or practice eg people living at altitude have higher Hb
Slide22Advantages
of guidelines
Can be useful education
Should include all evidenceShould improve overall standard of practiceCan be quoted in health economic assessments
Slide23Current efforts to produce guidelines
Are on going
Remain relatively region specific
Collaboration across EU in ELN and also includes USHow can MPN advocacy interact? In UK are involved.