

Available treatments for SCD
Pharmacologic treatments
Current available treatments mainly focus on managing acute symptoms and complications of sickle cell disease (SCD)
These treatments aim to reduce pain crises, reduce acute complications, or improve Hb levels.3-5
Red blood cell transfusions
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


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
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.