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Expert Highlights Diagnostic Challenges and Trial Access Barriers in Myeloproliferative Neoplasms

• Leading oncologist Dr. Ruben Mesa emphasizes that variable and common symptoms of myeloproliferative neoplasms, particularly fatigue and blood count-related issues, often lead to delayed diagnosis.

• Clinical trial recruitment faces significant hurdles due to limited availability at local centers, with trials primarily concentrated in larger academic institutions requiring extensive travel and resources.

• Health equity concerns in MPN clinical trials persist, as participation barriers disproportionately affect hourly workers and those with limited transportation or healthcare access.

Dr. Ruben Mesa, executive director of Atrium Health Wake Forest Baptist Comprehensive Cancer Center, has highlighted the complex challenges in diagnosing and conducting clinical trials for myeloproliferative neoplasms (MPNs), emphasizing the need for improved awareness and accessibility in treatment research.

Diagnostic Challenges in MPNs

The path to diagnosis for MPN patients often proves complicated due to the condition's nonspecific symptom profile. "The symptoms of myeloproliferative neoplasms are variable, can be problematic, but also are common, so it can be certainly nonspecific and not necessarily point people in the right direction to begin with," explains Dr. Mesa.
Myelofibrosis patients typically experience the most severe symptoms among MPN subtypes. The disease manifests through various indicators, with fatigue being the most prevalent. Other common symptoms include:
  • Headaches and difficulties with concentration
  • Migraines with aura
  • Enlarged spleen leading to early satiety and abdominal discomfort
  • Constitutional symptoms such as weight loss and night sweats
Diagnosis typically occurs through routine physical examinations, abnormal blood count detection, or following complications such as blood clots or bleeding events. Sometimes, an enlarged spleen is discovered incidentally through routine imaging.

Clinical Trial Access Barriers

The rarity of MPNs creates significant challenges in clinical trial recruitment and accessibility. Dr. Mesa points out that trials are predominantly available at larger academic centers with specialized hematology practices, limiting access for many patients.
To address these challenges, Dr. Mesa advocates for evolving trial designs that incorporate:
  • Telemedicine assessments for interim visits
  • Greater flexibility in enrollment criteria
  • Reduced frequency of in-person visits to registration centers

Health Equity Concerns in Clinical Research

The current clinical trial structure presents particular challenges for certain populations. Dr. Mesa emphasizes that participation often favors those with:
  • Comprehensive insurance coverage
  • Flexible employment arrangements
  • Substantial financial resources
  • Reliable transportation access
"The clinical trial process has historically exacerbated issues in health equity," Dr. Mesa notes, highlighting how the current system disadvantages hourly workers and those facing transportation challenges. This disparity in access continues to impact the diversity and representativeness of clinical trial populations in MPN research.

Advancing Solutions

The medical community is increasingly recognizing the need for more inclusive trial designs. Efforts are underway to expand trial accessibility through:
  • Implementation of hybrid visit models combining in-person and virtual care
  • Development of more localized trial sites
  • Creation of support systems for participants with limited resources
These initiatives aim to broaden participation while maintaining research integrity, ultimately working toward more equitable access to cutting-edge MPN treatments.
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Reference News

[1]
Recognizing Symptoms of Myeloproliferative Neoplasms and Clinical Trial Challenges
ajmc.com · Oct 24, 2024

Myeloproliferative neoplasms symptoms are variable and common, leading to delayed diagnoses. Clinical trials for these c...

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