Report on Anemia Management in Myelofibrosis: Emphasizing Sustainable Development Goals (SDGs)

Introduction

This report summarizes findings from an interview with Dr. Pankit Vachhani, Associate Professor of Medicine at the University of Alabama at Birmingham, conducted following the European Hematology Association 2025 Congress. The focus is on current treatment strategies for managing anemia in patients with myelofibrosis, highlighting unmet needs and clinical outcomes from recent research. The discussion aligns with the Sustainable Development Goals (SDGs), particularly SDG 3: Good Health and Well-being, aiming to improve health outcomes and promote innovative treatments.

Current Treatment Landscape for Anemia in Myelofibrosis

Anemia is a significant complication in myelofibrosis, a type of myeloproliferative neoplasm (MPN), affecting patients either at diagnosis or during treatment progression. The current therapeutic options include:

1. Four FDA-approved Janus kinase (JAK) inhibitors used in a line-agnostic manner:
   • Ruxolitinib (JAK1/JAK2 inhibitor, approved 2011)
   • Momelotinib (JAK1/JAK2 inhibitor with anemia management data)
   • Fedratinib
   • Pacritinib
2. Supportive care management strategies:
   • Erythropoietin-stimulating agents (ESAs) such as Procrit (Epoetin) or darbepoetin
   • Danazol
   • Blood transfusions
   • Luspatercept (under clinical investigation)

Despite these options, there is no drug specifically approved for anemia management in myelofibrosis, indicating a critical gap in treatment that aligns with SDG 3’s target to ensure access to quality essential medicines and vaccines.

Evidence Gaps and Ongoing Research

• Limited large-scale data exist on the efficacy of combination therapies, such as ruxolitinib with ESAs, danazol, or luspatercept.
• The INDEPENDENCE trial (NCT04717414), a phase 3 clinical study of luspatercept in transfusion-dependent patients on ruxolitinib, is expected to provide guidance by 2025.
• There is a need for new therapeutic agents that can simultaneously target the JAK-STAT pathway and support anemia management, promoting innovation in healthcare (SDG 9: Industry, Innovation, and Infrastructure).

Study Objectives and Methodology

The primary objective of Dr. Vachhani’s study was to evaluate treatment patterns and clinical outcomes in myelofibrosis patients receiving ruxolitinib combined with anemia-supporting medications (ESAs or danazol). This post-hoc analysis utilized data from the large phase 3b JUMP trial (NCT01493414), which enrolled 2,233 patients globally.

Study Design

• Participants: Patients diagnosed with primary or secondary myelofibrosis, with baseline platelet counts ≥50.
• Treatment: Ruxolitinib administered at 5 to 20 mg twice daily, dosage based on platelet count.
• Inclusion Criteria for Analysis:
  • Baseline anemia defined as hemoglobin
  • No ESA or danazol use within 3 months prior to enrollment.
  • Initiation of ESA or danazol within 3 months of enrollment and continuation for at least 3 months.
• Assessments:
  • Spleen length response
  • Symptom response using validated assessment tools

Alignment with Sustainable Development Goals

• SDG 3: Good Health and Well-being – The study contributes to improving treatment outcomes and quality of life for patients with myelofibrosis and anemia.
• SDG 9: Industry, Innovation, and Infrastructure – Emphasizes the need for innovative drug development and clinical trials to address unmet medical needs.
• SDG 17: Partnerships for the Goals – Highlights the importance of global collaboration in clinical research, as demonstrated by the multinational JUMP trial.

Conclusion

Current management of anemia in myelofibrosis involves multiple therapeutic strategies but lacks a dedicated anemia-specific drug, underscoring significant unmet needs. Ongoing clinical trials and post-hoc analyses of large datasets like the JUMP trial are essential to optimize treatment regimens and improve patient outcomes. These efforts support the achievement of key Sustainable Development Goals by advancing health innovation, ensuring access to effective treatments, and fostering international research partnerships.

1. Sustainable Development Goals (SDGs) Addressed or Connected to the Issues Highlighted in the Article

1. SDG 3: Good Health and Well-being
   • The article focuses on managing anemia in patients with myelofibrosis, a serious health condition.
   • It discusses current treatment options, unmet needs, and ongoing clinical trials aimed at improving patient outcomes.
   • Improving treatment and care for myelofibrosis patients directly contributes to ensuring healthy lives and promoting well-being for all ages.
2. SDG 9: Industry, Innovation, and Infrastructure
   • The article highlights the importance of clinical trials and drug development (e.g., JAK inhibitors, luspatercept) for advancing treatment options.
   • It emphasizes innovation in medical research and pharmaceutical development to address unmet medical needs.

2. Specific Targets Under Those SDGs Identified Based on the Article’s Content

1. SDG 3: Good Health and Well-being
   • Target 3.4: By 2030, reduce by one third premature mortality from non-communicable diseases through prevention and treatment and promote mental health and well-being.
   • Target 3.8: Achieve universal health coverage, including access to quality essential health-care services and access to safe, effective, quality, and affordable essential medicines and vaccines for all.
   • Target 3.b: Support the research and development of vaccines and medicines for the communicable and non-communicable diseases that primarily affect developing countries, provide access to affordable essential medicines and vaccines.
2. SDG 9: Industry, Innovation, and Infrastructure
   • Target 9.5: Enhance scientific research, upgrade the technological capabilities of industrial sectors, including encouraging innovation and substantially increasing the number of research and development workers.

3. Indicators Mentioned or Implied in the Article to Measure Progress Towards the Identified Targets

1. Clinical Outcomes and Treatment Efficacy Indicators
   • Spleen length response as a clinical measurement to assess treatment effectiveness.
   • Symptom response assessed using specific tools in clinical trials (e.g., JUMP trial assessments).
   • Hemoglobin levels to monitor anemia severity and response to anemia-supportive medications.
   • Use of transfusion requirements as an indicator of anemia management success.
2. Research and Development Indicators
   • Number and progress of clinical trials (e.g., INDEPENDENCE trial NCT04717414) evaluating new treatments.
   • Approval and availability of new drugs (e.g., JAK inhibitors, luspatercept) for myelofibrosis and anemia management.

4. Table of SDGs, Targets, and Indicators

Source: ajmc.com