Blood, Vol. 95 No. 10 (May 15), 2000:
pp. 3057-3064
Bone marrow transplantation versus periodic prophylactic blood
transfusion in sickle cell patients at high risk of ischemic
stroke: a decision analysis
Paul J. Nietert,
Miguel R. Abboud,
Marc D. Silverstein, and
Sherron M. Jackson
From the Center for Health Care Research and Departments of Medicine
and Pediatrics, Medical University of South Carolina, Charleston, SC.
Measurement of cerebral blood velocity (CBV) by transcranial Doppler
has been used to identify patients with sickle cell disease (SCD) who
are at high risk of ischemic stroke. This study examines outcomes of
bone marrow transplantation (BMT) and periodic blood transfusion (PBT)
as a basis for making treatment recommendations for patients who have
elevated CBV and no other indications for BMT. Decision analysis was
used to compare the number of quality-adjusted life years (QALYs)
experienced by a population of patients with SCD at high risk for
stroke who were treated with PBT or BMT. Markov models were constructed
to represent the clinical course of patients with SCD who were treated
with PBT or BMT. Medical literature and expert opinion provided risks
of stroke and death for different disease states, estimates of
transition probabilities from one clinical state to another, and
quality of life. An intention-to-treat analysis and an analysis of
treatment received were both performed on hypothetical cohorts of
100 000 patients. Patients with SCD who were managed with a strategy
of intending to provide BMT could expect 16.0 QALYs, compared with 15.7 QALYs for a strategy of intending to provide PBT; however, the
variation around these estimates was large. In the treatment received
analysis, patients compliant with PBT therapy and iron chelation could
expect the best outcomes (19.2 QALYs). From a policy perspective,
neither BMT nor PBT can be considered the "best" treatment for
children with SCD who have abnormal CBV. Abnormal CBV should not be the only criterion for selecting patients with sickle cell for BMT.
