Improved cerebrovascular patency following therapy in patients with sicklecell disease: Initial results in 4 patients who received HLA-identical hematopoietic stem cell allografts

To test whether magnetic resonance angiography can document the evolution o f vasculopathy in patients with sickle cell disease, we reviewed records to identify all patients who underwent magnetic resonance angiography from 19 93 to 1999. Of 512 angiographies performed, 105 were of sickle cell disease patients, and 24 sickle cell disease patients 7 years of age or older unde rwent baseline and follow-up examinations. Films were paired by patient, bl inded as to examination date and treatment, and quantitatively compared. Fo ur patients who received allogeneic bone marrow transplantation were compar ed to 7 patients who received other therapy and to 13 untreated patients. Q uantitative analysis revealed a 10% increase in the measured diameter of 64 vessels (p = 0.001) following any treatment. Patients who had undergone al logeneic bone marrow transplantation exhibited a 12% increase in the lumen of 22 vessels (p = 0.041), whereas patients treated with chronic transfusio n or hydroxyurea exhibited an 8% increase in 42 vessels (p = 0.016). In 2 p atients with severe stenosis, the artery normalized after transplantation, and the blood flow rate was reduced in all patients who underwent transplan tation. In untreated patients, there was a trend for the size of the arteri al lumen to decrease, which is consistent with disease progression. Results suggest that treatment can reverse progression of vasculopathy. Bone marro w transplantation may enable stenoses to heal and can substantially reduce cranial blood velocity, suggesting that allogeneic bone marrow transplantat ion may prevent infarction or brain damage.