Available treatments for SCD

Pharmacologic treatments

Current available treatments mainly focus on managing acute symptoms and complications of sickle cell disease (SCD)

Treatments are available that can reduce the frequency of pain crises and the need for blood transfusions.¹ In recent years, several treatments have been approved by the FDA for the treatment of SCD, representing treatment advances that haven't occurred in decades.^2-5

These treatments aim to reduce pain crises, reduce acute complications, or improve Hb levels.^3-5

Red blood cell transfusions

Chronic red blood cell transfusions aim to reduce the proportion of HbS in total Hb and may effectively prevent stroke and decrease the frequency of pain events and severity of acute chest syndrome.⁶

Patients on chronic transfusions may receive chelation therapy to manage iron levels; however, iron overload can still occur in multiple organs and iron overload–induced organ damage has the potential to become fatal.^7,8

Allogeneic HSCT

Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative option for treatment of SCD; however, its availability remains limited and transplant decisions are generally based on a patient's benefit versus risk and donor availability.⁹

There are inherent risks associated with allogeneic HSCT, including⁹:

Transplant-related mortality
Severe immunological complications, such as graft versus host disease (GVHD)
Graft rejection
Infection

Treatment opportunities

Advances in therapies for SCD, including recently approved treatments, have helped to provide additional options for healthcare professionals to treat patients with SCD.¹ However, there remains a need for broadly accessible treatments that can prevent or eliminate lifelong symptoms, complications, and associated comorbidities that result from SCD. Ultimately, such treatments may have the potential to impact the future of this disease.

Abena O. Appiah-Kubi, MD, discussing partnering with patients

Despite all the complications sickle cell disease can cause, our role and our goal is to partner with patients to try to prevent as much from happening as we can. The future looks bright.”

Abena O. Appiah-Kubi, MD, MPH

New therapies are being investigated that focus on treating
SCD at the genetic level New therapies are being investigated that focus on treating SCD at the genetic level

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References

1. Kato GJ, Piel FB, Reid CD, et al. Sickle cell disease. Nat Rev Dis Primers. 2018;4:18010. 2. Kapoor S, Little JA, Pecker LH. Advances in the treatment of sickle cell disease. Mayo Clin Proc. 2018;93(12):1810-1824. 3. ENDARI™ (L-glutamine oral powder) [package insert]. Torrance, CA: Emmaus Medical, Inc; 2017. 4. ADAKVEO^® (crizanlizumab) [package insert]. East Hanover, NJ: Novartis Pharmaceutical Corporation; 2019. 5. OXBRYTA™ (voxelotor) [package insert]. San Francisco, CA: Blood Therapeutics, Inc; 2019. 6. Ware RE, de Montalembert M, Tshilolo L, Abboud MR. Sickle cell disease. Lancet. 2017;390(10091):311-323. 7. de Montalembert M, Ferster A, Colombatti R, Rees DC, Gulbis B; European Network for Rare and Congenital Anaemias. ENERCA clinical recommendations for disease management and prevention of complications of sickle cell disease in children. Am J Hematol. 2011;86(1):72-75. 8. Wood JC, Cohen AR, Pressel SL, et al. Organ iron accumulation in chronically transfused children with sickle cell anaemia: baseline results from the TWiTCH trial. Br J Haematol. 2016;172(1):122‑130. 9. Meier ER, Abraham A, Fasano RM, eds. Sickle Cell Disease and Hematopoietic Stem Cell Transplantation. Springer International Publishing; 2018.