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Sickle Cell Disease: Treatment Options of the Past, Present, and Future

Charleen T Jacobs-McFarlane¹, Angela Liu

¹Charleen T. Jacobs-McFarlane is an NP at Mount Sinai Hospital, and Angela Liu is a hematologist at the Icahn School of Medicine at Mount Sinai, both in New York City. Jacobs-McFarlane has reported receiving ongoing funding from the National Heart, Lung, and Blood Institute, National Institutes of Health, and past funding from Pfizer. Contact author: Charleen T. Jacobs-McFarlane, charleen.jacobs-mcfarlane@mountsinai.org . The authors and planners have disclosed no potential conflicts of interest, financial or otherwise.

The American Journal of Nursing

|December 24, 2025

Insights

Sickle cell disease (SCD) is a serious inherited blood disorder. Recent advances offer new treatments and hope for improved patient outcomes, with nurses playing a key role in care.

Area of Science:

Hematology
Genetics
Public Health

Background:

Sickle cell disease (SCD) affects 70,000-100,000 individuals in the US, primarily of African descent.
SCD causes significant pain, end-organ damage, high morbidity, and early mortality.
Despite challenges, research and treatment advancements are improving patient outcomes.

Purpose of the Study:

To review historical perspectives, current treatments, and future directions for SCD care.
To highlight the evolving landscape of SCD therapies.
To emphasize the role of nurses in advancing SCD care.

Main Methods:

Literature review of historical data and recent advancements in SCD research and treatment.
Analysis of FDA-approved medications and therapies for SCD.
Discussion of the current and future roles of healthcare professionals, particularly nurses.

Main Results:

Several new medications and curative therapies for SCD have been FDA-approved in the last decade.
A robust pipeline of novel SCD treatments is under development.
Nurses are crucial for patient education, research, advocacy, and implementing new therapies.

Conclusions:

Advances in research and treatment have significantly improved outcomes for individuals with SCD.
The field of SCD care is rapidly evolving with promising new therapeutic options.
Nurses are integral to the successful implementation of emerging SCD therapies and overall patient care.

Read full analysis

Abstract:

Sickle cell disease (SCD) is an inherited red blood cell disorder that affects an estimated 70,000 to 100,000 people in the United States, most of whom are of African descent. SCD causes acute and chronic pain and widespread end-organ damage, resulting in acute and chronic complications, high morbidity, and early death. Advances in research and treatment have improved patient outcomes for people living with SCD. Over the past 10 years, several new medications and curative therapies have been approved by the U.S. Food and Drug Administration for people with SCD, and more medications are in the pipeline. This review includes historical perspectives, current treatment options, and future directions for this population. With the ongoing rapid changes in SCD care, nurses are well positioned to play a role in patient education, research, advocacy, and the implementation of these emerging therapies to advance the care of those affected by SCD.

Keywords:

access to care clinical updates curative therapies sickle cell disease

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