touchPANEL DISCUSSION

Tailoring treatment in myelofibrosis: Addressing anaemia and thrombocytopenia

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Poll

In your opinion what is the most important strategy to maximize the success of JAK inhibitor treatment in your patients with myelofibrosis?

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Patient education
   
Regular monitoring for treatment response
   
Early treatment initiation
   
Other
   

Tutorial

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Poll

What is your most important consideration when selecting a JAK inhibitor for your patients with myelofibrosis and cytopenias?

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Patients’ comorbidities
   
Impact on cytopenias
   
Tolerability and side effects
   
Other
   

Tutorial

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Poll

What is the biggest challenge for your patients with myelofibrosis and cytopenias?

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Fatigue and physical strain
   
The burden of transfusions
   
The impact on QoL
   
Other
   
 
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Myeloproliferative Disorders, Anaemia, Rare Diseases, Thrombocytopenia CE/CME accredited

touchPANEL DISCUSSION
A visually engaging discussion designed to emulate a ā€˜live’ panel experience and provide clinicians with practical expert insights to address their clinical challenges. Useful tips below will show how to navigate the activity. Close

Tailoring treatment in myelofibrosis: Addressing anaemia and thrombocytopenia

  • Select in the video player controls bar to choose subtitle language. Subtitles available in English, French, German, Italian, Japanese, Portuguese, Spanish.
  • A practice aid is available for this activity in the Toolkit
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Learning Objectives

After watching this activity, participants should be better able to:

  • Evaluate the burden of disease and QoL impact for patients with myelofibrosis and anaemia and/or thrombocytopenia and the importance of early treatment initiation
  • Assess the current management strategies for patients with myelofibrosis and anaemia and/or thrombocytopenia and the impact on patient outcomes, including QoL
  • Review the emerging treatment options for managing patients with myelofibrosis, and other strategies to optimize outcomes
Overview

In this activity, three leading experts discuss the burden of myelofibrosis and anaemia and/or thrombocytopenia before reviewing approved and emerging targeted treatment options and strategies to maximize benefits for patients. read more

Target Audience

This activity has been designed to meet the educational needs of haematologists, including haemato-oncologists, hospital pharmacists and haematology nurses involved in the management of patients with myelofibrosis and anaemia and/or thrombocytopenia.

EBACĀ® Accreditation

touchIME is an EBACĀ® accredited provider since 2023.

This programme is accredited by the European Board for Accreditation of Continuing Education for Health Professionals (EBACĀ®) for 49 minutes of effective education time.

The Accreditation Council for Continuing Medical Education (ACCME) and the Royal College of Physicians and Surgeons of Canada hold an agreement on mutual recognition on substantive equivalency of accreditation systems with EBACĀ®.

Through an agreement between the European Board for Accreditation of Continuing Education for Health Professionals and the American Medical Association (AMA), physicians may convert EBACĀ® CE creditsĀ to AMA PRA Category 1 CreditsTM. Information on the process to convert EBACĀ® credit to AMA credit can be found on the AMA website. Other healthcare professionals may obtain from the AMA a certificate of having participated in an activity eligible for conversion of credit to AMA PRA Category 1 CreditTM.

Faculty Disclosure Statement / Conflict of Interest Policy

In compliance with EBACĀ® guidelines, all speakers/chairpersons participating in this programme have disclosed or indicated potential conflicts of interest, which might cause a bias in the presentations. The Organizing Committee/Course Director is responsible for ensuring that all potential conflicts of interest relevant to the event have been mitigated and declared to the audience prior to the CME activities.

Faculty

Prof. Haifa Kathrin Al-Ali discloses: Advisory board or panel fees from AOP Pharma, Bristol Myers Squibb, GSK, Incyte, MSD and Novartis. Consultant fees from AbbVie, AOP Pharma, Blueprint, Bristol Myers Squibb, GSK, MSD, Novartis, Otsuka and Stemline. Grants/research support from Bristol Myers Squibb and Incyte. Other financial or material support (royalties, patent, etc.) from AbbVie and Alexion.

Prof. Jean-Jacques Kiladjian discloses: Advisory board or panel fees from AOP Health and Incyte. Consultant fees from AbbVie, Bristol Myers Squibb, GSK and Novartis.

Prof. Alessandro Vannucchi discloses: Advisory board or panel fees from AbbVie, AOP Orphan Pharmaceuticals, Blueprint, Bristol Myers Squibb, Celgene, Constellation, CTI BioPharma, Geron, GSK, Imago, Incyte, iOnctura, Italfarmaco, Kartos, Karyopharm, Keros, MorphoSys, Novartis, Protagonist, Roche and Sierra.

Touch Medical Contributors

Katrina Lester has no financial interests/relationships or affiliations in relation to this activity.

Requirements for Successful Completion

Certificates of Completion may be awarded upon successful completion of the post-test and evaluation form. If you have completed one hour or more of effective education through EBACĀ® accredited CE activities, please contact us at accreditation@touchime.org to receive your EBACĀ® CE credit certificate. EBACĀ® grants 1 CE credit for every hour of education completed.

Date of original release: 14 April 2025. Date credits expire: 14 April 2027.

Time to complete: 49 minutes

If you have any questions regarding the EBACĀ® credits, please contact accreditation@touchime.orgĀ Ā 

Ā 

This activity is CE/CME accredited

To obtain the CE/CME credit(s) from this activity, please complete this post-activity test.

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  • Select in the video player controls bar to choose subtitle language. Subtitles available in English, French, German, Italian, Japanese, Portuguese, Spanish.
  • A practice aid is available for this activity in the Toolkit
  • Downloads including slides are available for this activity in the Toolkit

Topics covered in this activity

Myeloproliferative Disorders / Anaemia / Rare Diseases / Thrombocytopenia
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touchPANEL DISCUSSION
Tailoring treatment in myelofibrosis: Addressing anaemia and thrombocytopenia
0.75 CE/CME credit

Question 1/5
In a global study that investigated the QoL impact of anaemia and transfusion dependence in patients with symptomatic MF, what percentage of patients reported frequent transfusions as inconvenient?

MF, myelofibrosis; QoL, quality of life.

A multinational, cross-sectional, online survey study was carried out between October 2023 and February 2024 in patients with MF from the US, UK, Germany, Italy, Spain, and Poland (N=155) to assess the associated QoL impact of anaemia and transfusion dependence. Patients were symptomatic and previously treated with a JAK inhibitor. Overall, 59% of patients reported frequent transfusions to be “extremely” or “quite a bit” inconvenient. The most common inconveniences reported were time spent at the clinic (81%), travel time (40%) and scheduling issues (32%).

Abbreviations
JAK, Janus kinase; MF, myelofibrosis; QoL, quality of life.

Reference
LeBlanc TW, et al. Blood. 2024;144(Suppl. 1):3815.

Question 2/5
Your patient is diagnosed with symptomatic MF and anaemia. You recommend starting treatment with a JAK inhibitor. Your patient asks about expected outcomes associated with treatment. In addition to the reported improvements in constitutional symptoms and splenomegaly, what might you tell them about JAK inhibitor treatment?

JAK, Janus kinase; MF, myelofibrosis.

The JAK inhibitors ruxolitinib and fedratinib improve constitutional symptoms and splenomegaly, but carry on-target risks of worsening anaemia and thrombocytopenia, limiting their use in patients with cytopenic MF.1

Momelotinib is approved for disease-related splenomegaly or symptoms in adults with moderate-to-severe anaemia and primary MF, PPV-MF or PET-MF.2 Post hoc analyses of data from the pivotal phase III SIMPLIFY-1, -2 and MOMENTUM trials showed that relative to ruxolitinib, momelotinib was an effective treatment for patients with MF and moderate-to-severe thrombocytopenia.3 Pacritinib is FDA-approved for adults with intermediate or high-risk primary or secondary MF with a PC <50 x 109/L,4 and has demonstrated spleen and symptom reduction in patients with MF regardless of cytopenias.5

Abbreviations
FDA, US Food and Drug Administration; JAK, Janus kinase; MF, myelofibrosis; PC, platelet count; PET, post-essential thrombocythaemia; PPV, post-polycythaemia vera.

References

  1. Reynolds SB, Pettit K. Hematology Am Soc Hematol Educ Program. 2022;2022:235–44.
  2. EMA. Momelotinib SmPC. Available at: https://bit.ly/4c5jzAE (accessed 27 March 2025).
  3. Kiladjian J-J, et al. Hemasphere. 2023;7:e963.
  4. FDA. Pacritinib PI. Available at: https://bit.ly/4j3auKK (accessed 27 March 2025).
  5. Bose P, et al. J Clin Oncol. 2023;41(Suppl. 16):7068.
Question 3/5
Your patient was diagnosed with high-risk primary MF and mild anaemia (Hb 9.8 g/dL) and thrombocytopenia (PC 120 x 109/L). Ruxolitinib treatment demonstrated an initial spleen response, but after 8 months of treatment, your patient developed transfusion-dependent anaemia (Hb 7.0 g/dL) and worsening thrombocytopenia (PC 58 x 109/L). Their ruxolitinib dose was reduced accordingly. Three months later, your patient reports persistent fatigue and splenic discomfort (palpable 10 cm below LCM); PC 49 x 109/L and Hb 7.9 g/dL. What would you recommend to best manage this patient?

ESA, erythropoiesis-stimulating agent; Hb, haemoglobin; LCM, left costal margin; MF, myelofibrosis; PC, platelet count.

Momelotinib represents a tolerable and effective treatment option for patients with moderate-to-severe anaemia and primary MF, as well as in patients with moderate-to-severe thrombocytopenia.1,2 Patients with MF and a PC <50 x 109/L should not be treated with ruxolitinib or fedratinib.3,4

Abbreviations
MF, myelofibrosis; PC, platelet count.

References

  1. EMA. Momelotinib SmPC. Available at: https://bit.ly/4c5jzAE (accessed 27 March 2025).
  2. Kiladjian J-J, et al. Hemasphere. 2023;7:e963.
  3. EMA. Ruxolitinib SmPC. Available at: https://bit.ly/41UP8Zi (accessed 27 March 2025).
  4. EMA. Fedratinib SmPC. Available at: https://bit.ly/4iYRkpx (accessed 27 March 2025).
Question 4/5
In a retrospective real-world study in patients with MF in the United States, what was the 1-year OS probability associated with pacritinib treatment?

MF, myelofibrosis; OS, overall survival.

In a study assessing real-world treatment patterns and outcomes in patients with MF treated with pacritinib in the United States, 1-year OS probability was 69.4%.1 Pacritinib is FDA-approved for patients with intermediate or high-risk primary or secondary MF and a PC <50 x 109/L.2 It is in phase III development in Europe and other countries.3

Abbreviations
FDA, US Food and Drug Administration; MF, myelofibrosis; OS, overall survival; PC, platelet count.

References

  1. Marrone M, et al. J Clin Oncol. 2024;42(Suppl. 16):6579.
  2. FDA. Pacritinib PI. Available at: https://bit.ly/4j3auKK (accessed 27 March 2025).
  3. ClinicalTrials.gov. NCT03165734. Available at: https://bit.ly/4i2rCzL (accessed 27 March 2025).
Question 5/5
You have initiated JAK inhibitor treatment in your patient with MF. How would you best monitor for treatment response to inform the need for potential changes to their treatment plan?

JAK, Janus kinase; LCM, left costal margin; MF, myelofibrosis; PC, platelet count; PRO, patient-reported outcome; WBC, white blood cell.

According to recommendations from a global consensus group, response to JAK inhibitor treatment in patients with MF should be measured every 3–6 months depending on MF risk category and stability. Assessments should include, but are not limited to, accurately determined spleen size, e.g. using a measuring tape; and assessing symptom score with a validated tool, e.g. MPN-SAFTSS or MFSAF.

Abbreviations
JAK, Janus kinase; MF, myelofibrosis; MFSAF; MF Symptom Assessment Form; MPN-SAFTSS, Myeloproliferative Neoplasm SAF Total Symptom Score.

Reference
Koschmieder S, et al. Leukemia. 2024;38:1831–8.

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