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Sickle Cell Disease: A Brief Update.

Sharl Azar1, Trisha E Wong2

  • 1Division of Hematology and Medical Oncology, Department of Medicine, Oregon Health and Science University, 3181 Southwest Sam Jackson Park Road, Mailstop L586, Portland, OR 97239, USA.

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|February 13, 2017
PubMed
Summary
This summary is machine-generated.

Sickle cell disease (SCD) is a genetic disorder affecting red blood cells, leading to severe health issues. While a cure is limited, promising new therapies are emerging for this growing global health concern.

Keywords:
HemoglobinopathyHydroxyureaIron overloadReviewSickle cell anemiaSickle cell disease

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Area of Science:

  • Genetics and Hematology
  • Inherited Blood Disorders
  • Monogenic Diseases

Background:

  • Sickle cell disease (SCD) is an inherited monogenic disorder.
  • Characterized by misshapen red blood cells, leading to vaso-occlusive disease, vasculopathy, and systemic inflammation.
  • Affects approximately 300,000 infants globally each year, causing significant end-organ damage and reduced quality of life.

Purpose of the Study:

  • To summarize the current understanding of sickle cell disease.
  • To highlight the limitations of existing treatments like hematopoietic stem cell transplantation.
  • To underscore the hope offered by emerging therapies in late-stage clinical trials.

Main Methods:

  • Review of existing literature on sickle cell disease.
  • Analysis of global incidence and impact.
  • Overview of current and pipeline therapeutic strategies.

Main Results:

  • Sickle cell disease presents with severe acute and chronic complications.
  • Hematopoietic stem cell transplantation is the only current cure but has limited accessibility.
  • Numerous novel therapies are progressing through clinical trials.

Conclusions:

  • Sickle cell disease poses a significant and increasing global health burden.
  • The lack of broadly accessible cures necessitates the development of new treatments.
  • Advancements in therapeutic pipelines offer substantial hope for patients with SCD.