Hydroxyurea and Its Role in Sickle Cell Disease During Pregnancy

Hydroxyurea has long been considered a controversial treatment during pregnancy for patients with sickle cell disease (SCD), particularly due to fears of potential harm to the developing fetus. However, recent findings presented at the American Society of Hematology (ASH) 2025 Annual Meeting provide new insights that challenge these preconceptions.

Dr. Anoosha Habibi from the Sickle Cell Referral Center in France reported that post-marketing safety data indicates hydroxyurea can be continued safely during pregnancy without increased risks for maternal health or substantial teratogenic malformations in newborns. This assessment comes from a study involving over 200 patients in the European Sickle Cell Disease Cohort-HydroxyUrea (ESCORT-HU) and its extension study, during which 245 pregnancies occurred with a significant portion involving mothers who were either on hydroxyurea or who had recently used the medication.

In her presentation, Habibi highlighted that no maternal deaths were recorded, and the instances of birth defects were minimal. This data significantly alters the previous narrative that highlighted the potential dangers of hydroxyurea in pregnant patients, especially in low-resource environments where blood transfusions may not be a viable alternative.

Paving the Way for Better Clinical Decisions

The implications of these findings extend beyond reassurance for patients; they also offer a pivotal opportunity for healthcare professionals to rethink treatment protocols. Historically, the standard advice for women taking hydroxyurea has been to discontinue the medication several months before conception to mitigate any potential risks to the fetus. However, given that hydroxyurea is reported to significantly reduce mortality in SCD patients, this new evidence suggests that continuing treatment could be critical for the mother's health—a decision that should be considered carefully on a case-by-case basis.

Understanding Accidental Exposure and Its Implications

One of the key takeaways from the studies was the potential risks associated with accidental exposure to hydroxyurea during early pregnancy. Many patients find themselves unknowingly pregnant while on hydroxyurea, often leading to anxiety over potential harm. The reassurance provided by the recent findings is invaluable in simplifying the decisions faced by patients and their healthcare teams. Instead of simply discontinuing treatment and risking adverse SCD-related complications, this information encourages a more balanced view of the potential outcomes.

Collaborating for Better Outcomes in Under-Resourced Areas

In low-resource settings, where transfusions may not be readily available, the existence of hydroxyurea becomes even more critical. The ongoing challenge has been making informed clinical decisions that weigh the benefits of treatment against the historical fears surrounding teratogenic risks. Dr. Habibi’s research underscores that in some cases, continuing hydroxyurea may present a lower risk than discontinuing it, especially in areas with limited medical resources.

The Broader Picture: Historical Context and Future Investigations

Previous studies have provided conflicting perspectives, with some highlighting potential risks while others focused on the benefits of hydroxyurea. Thus, ongoing research and wider clinical trials are necessary to further clarify these uncertainties. The ASH and other organizations are urged to establish clinical guidelines that incorporate the latest findings, ensuring that healthcare providers are equipped with up-to-date information when advising patients. Enhancing understanding of hydroxyurea’s safety during pregnancy can ultimately pave the way for improved maternal health outcomes and encourage informed patient participation in their treatment decisions.

Conclusion: Taking Action with New Information

As research evolves, so too does our responsibility to stay informed. For patients grappling with sickle cell disease and considering pregnancy, these recent findings offer a sense of hope and renewed trust in treatment protocols. By engaging healthcare professionals in discussions about the best path forward—grounded in safety but also in the continued need for treatment—patients can feel empowered to make educated decisions about their health.

Continued education and awareness can lead to better healthcare practices. Stay informed and connected with healthcare providers to ensure comprehensive and safe management of sickle cell disease during pregnancy.