At a population level, iron deficiency and IDA remain widespread global health issues, says George Goshua, MD, MSc, assistant professor of medicine (medical oncology and hematology) at Yale School of Medicine.
“Collectively, we in medicine have had little advancement in addressing iron deficiency and iron deficiency anemia, affecting up to 2 billion and 1.2 billion women respectively, globally, dating back to at least 2000 now,” he says.
Treating IDA in the setting of heavy menstrual bleeding typically involves iron supplementation, which can help alleviate symptoms and restore iron levels while the underlying cause of bleeding is addressed. Iron can be administered orally or intravenously, with IV formulations differing in dosing schedules, some delivered as a single high-dose infusion and others as multiple smaller doses over time.
In clinical practice, treatment decisions are often shaped by a mix of effectiveness, tolerability, and access, says Daniel Wang, a medical student in Goshua’s lab.
“Oral iron is often the first option patients receive, but it can be difficult to tolerate because of gastrointestinal side effects and may not be as effective when blood loss is ongoing,” Wang says. “IV iron, on the other hand, introduces a different set of challenges, including insurance restrictions and the need for repeated clinic visits.”
These real-world tradeoffs, which can result in delayed treatment and inadequate iron repletion, inspired Wang to investigate how different iron formulations compare, not only in terms of clinical effectiveness, but also in quality of life, treatment burden, and cost over time.
Wang and his team built a decision-analytic Markov model to simulate patients’ treatment courses across thousands of scenarios.
“Our Markov model is a statistical tool that’s commonly used in decision science, and it helps us quantify outcomes when there’s uncertainty,” says Wang. “You can run the model over thousands of iterations, with each one simulating a patient’s walk through different health states.”
The model draws on data from existing literature and real-world evidence, incorporating what is known about patients’ experiences, iron metabolism, and differences in iron absorption between oral and IV therapies. “In that sense,” Wang adds, “it almost lets you run a randomized trial without actually running one, which would be incredibly difficult to do at that scale.”
Across a wide range of scenarios in the model, the team identified IV iron, specifically the formulation IV iron dextran, as the most cost-effective first-line treatment.
“IV iron dextran can be dosed as a very large, one-time infusion,” says Wang. “At face value, IV iron looks more expensive, but when you look over the long run, especially for women with ongoing blood loss, it provides much better value. That includes not just what insurers pay, but the time and costs patients absorb themselves, like childcare, transportation, and taking off work.”
The study offers a more patient-centered framework for evaluating first-line therapy for IDA, particularly for women with heavy menstrual bleeding, who often cycle through treatment delays and suboptimal care.
“For women with iron deficiency, with or without anemia, whose values and preferences align with IV iron treatment, they need to have that option,” Goshua emphasizes. “And it needs to be in the form of a convenient one-time infusion so they can tend to the rest of their life with minimal disruptions for an easily fixable issue.”
Goshua hopes the findings will help shift how clinicians, health systems, and insurers think about treatment decisions in this population.
“This study is one step in the right direction of work that, thanks to the American Society of Hematology, many stakeholders have started improving for women in the U.S. and globally,” he says.
Medical Oncology and Hematology, one of 10 sections in the Yale Department of Internal Medicine, engages in comprehensive and innovative patient care, research, and educational activities for a broad range of oncologic and hematologic diseases. To learn more, visit Medical Oncology and Hematology.