TABLE 13Characteristics of non-randomised studies

Study name and designIntervention and dose (n)OutcomesInclusion criteriaExclusion criteriaFollow-up, months: mean (SD)AEResults
Mirre (2010)68

Retrospective cohort study, single centred, France, n = 17
Blood transfusion (n = 17)

To reach target HbS concentration of < 30% of total haemoglobin without exceeding haemoglobin concentration of 12g/dl

Once HbS > 30%, transfusion every 4 weeks

Transfusion type at discretion of investigator
Primary:
  • Success/failure in preventing stroke
Secondary:
  • Assess clinical/haematological/neuroimaging changes and complications following chronic transfusion
  • Diagnosis of SCA or HbSβ0 thalassaemia
  • ≥ 2–16 years old
  • ≤ 2 abnormal TCD ultrasonography readings (time averaged mean blood velocity ≤ 200 cm/second)
  • NR
32.4 (20.4)Hepatitis B 0/17; Hepatitis C 0/17; bone marrow transplant 2/17; increased TCD velocity 2/17; chelation NR; alloimmunisation NRStroke 0/17

NR, not reported.

From: 4, Assessment of clinical effectiveness

Cover of The Clinical Effectiveness and Cost-Effectiveness of Primary Stroke Prevention in Children with Sickle Cell Disease: A Systematic Review and Economic Evaluation
The Clinical Effectiveness and Cost-Effectiveness of Primary Stroke Prevention in Children with Sickle Cell Disease: A Systematic Review and Economic Evaluation.
Health Technology Assessment, No. 16.43.
Cherry MG, Greenhalgh J, Osipenko L, et al.
© 2012, Crown Copyright.

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