Sickle Cell Disease (Sickle Cell Anemia): Therapy

General preventive measures to protect against sickle cell crisis

  • Avoidance of
    • Desiccosis (dehydration)
    • Infections
    • Hypothermia
    • Oxygen deficiency conditions – altitude from 2,000 m; flights.
  • Nicotine restriction (refraining from tobacco use).
  • Alcohol restriction (abstaining from alcohol)
  • Review of permanent medication due topossible effect on the existing disease.

Further prophylactic measure

General therapeutic measures

  • Bloodletting – indicated in cases of high blood viscosity (viscosity of blood) associated with frequent pain crises

Causal therapy

  • Allogeneic stem cell transplantation (more precisely, hematopoietic stem cell transplantation; HSCT; blood stem cell transplantation) – transfer of stem cells from an individual of the same species with different genetics (family donor):
    • In children under 16 years of age, complete allogeneic stem cell therapy is now the goal. Most patients can even discontinue immunosuppressants (drugs that reduce immune system functions) after some time without the dreaded graft-versus-host (graft-host) reaction.
    • For elderly patients, the concept of a “mini” stem cell transplant has been developed. As a result, these patients then have both erythrocytes (red blood cells) that are prone to sickle cell formation due to the genetic defect and healthy erythrocytes. The therapeutic success is shown by the increase of the patients’ hemoglobin (to well over 10 g/dl). The disadvantage of this form of therapy is that patients must take immunosuppressants.
  • Gene therapy: infection of hematopoietic stem cells with a virus (lentiviruses) that deposited the genetic information for a correct version of the gene in the cells; preparation of the patient with the alkylane busulfan; the treated patient – now 15 years old – has remained without further sickle cell crises for 15 months.

Vaccinations

The following vaccinations are advised: