Transplantation of blood-forming stem cells for children with sickle cell disease

Review question

We reviewed the evidence about the cure rate and risks of hematopoietic stem cell transplantation for people with sickle cell disease.

Background

Sickle cell disease is a genetic disorder mainly characterized by the presence of deformed, sickle-shaped red blood cells in the blood stream. These cells deprive tissues of blood and oxygen resulting in periodic and recurrent painful attacks. Complications include acute chest syndrome and stroke. Although sickle cell disease is responsive to preventive and supportive measures such as the use of prophylactic antibodies and periodic blood transfusion, these do not provide a cure. The use of hematopoietic (blood forming) stem cell transplantation involves replacing the deformed red blood cells and its stem cells with stem cells from a healthy donor thereby producing normal red blood cells. These stem cells can be derived from either the bone marrow or blood (umbilical cord blood or peripheral blood) of a healthy individual. This is an update of a previously published review.

Search date

The evidence is current to: October 2015.

Key results

There are no randomized controlled trials assessing the benefits and risks; the most appropriate source of stem cells; or the most eligible participants (those who have experience severe complication or those who have not) of the procedure in people with sickle cell disease.

Authors' conclusions: 

Reports on the use of hematopoietic stem cell transplantation improving survival and preventing symptoms and complications associated with sickle cell disease are currently limited to observational and other less robust studies. No randomized controlled trial assessing the benefit or risk of hematopoietic stem cell transplantations was found. Thus, this systematic review identifies the need for a multicentre randomized controlled trial assessing the benefits and possible risks of hematopoietic stem cell transplantations comparing sickle status and severity of disease in people with sickle cell disease.

Read the full abstract...
Background: 

Sickle cell disease is a genetic disorder involving a defect in the red blood cells due to its sickled hemoglobin. The main therapeutic interventions include preventive and supportive measures. Hematopoietic stem cell transplantations are carried out with the aim of replacing the defective cells and their progenitors (hematopoietic (i.e. blood forming) stem cells) in order to correct the disorder. This is an update of a previously published review.

Objectives: 

To determine whether stem cell transplantation can improve survival and prevent symptoms and complications associated with sickle cell disease. To examine the risks of stem cell transplantation against the potential long-term gain for people with sickle cell disease.

Search strategy: 

We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Group's Haemoglobinopathies Trials Register complied from electronic searches of the Cochrane Central Register of Controlled Trials (CENTRAL) (updated each new issue of The Cochrane Library) and quarterly searches of MEDLINE.

Unpublished work was identified by searching the abstract books of major conference proceedings and we conducted a search of the website: www.ClinicalTrials.gov.

Date of the most recent search of the Group's Haemoglobinopathies Trials Register: 06 October 2015.

Selection criteria: 

Randomized controlled and quasi-randomized studies that compared any method of stem cell transplantation with either each other or with any of the preventive or supportive interventions (e.g. periodic blood transfusion, use of hydroxyurea, antibiotics, pain relievers, supplemental oxygen) in people with sickle cell disease irrespective of the type of sickle cell disease, gender and setting.

Data collection and analysis: 

No relevant trials were identified.

Main results: 

Ten trials were identified by the initial search and none for the update. None of these trials were suitable for inclusion in this review.

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