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Related Experiment Video

Updated: Mar 16, 2026

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Sickle cell disease.

Raffaella Colombatti1, Wasil Jastaniah2, Julie Makani3

  • 1Department of Woman's and Child's Health, University of Padova, Padova, Italy; Pediatric Hematology-Oncology Unit, Azienda Ospedale-Università di Padova, Padova, Italy.

Lancet (London, England)
|March 14, 2026
PubMed
Summary
This summary is machine-generated.

Sickle cell disease (SCD) is a complex genetic disorder. Recent advances in treatments like gene therapy and hydroxyurea offer new hope for managing SCD complications and improving patient outcomes globally.

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Area of Science:

  • Hematology
  • Genetics
  • Internal Medicine

Background:

  • Sickle cell disease (SCD) is a prevalent genetic red blood cell disorder with significant global impact.
  • The disease involves complex pathophysiology including sickle hemoglobin polymerization, vaso-occlusion, hemolysis, and inflammation, leading to severe complications.

Purpose of the Study:

  • To provide a comprehensive update on sickle cell disease.
  • To review current and emerging therapeutic strategies and implementation challenges.

Main Methods:

  • Review of recent scientific literature and clinical trial data.
  • Analysis of global implementation strategies in various healthcare settings.

Main Results:

  • Significant advances include expanded hydroxyurea use, refined transfusion therapy, improved stem cell transplantation, and approved gene therapies.
  • New drugs are under global clinical evaluation, with successful implementation models in low- and middle-income countries.

Conclusions:

  • Current advancements offer improved management options for sickle cell disease.
  • Addressing challenges in specific variants, prevention, and care transition is crucial for comprehensive SCD management.