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Published on: March 14, 2017
Improved stenosis outcome in stroke-free sickle cell anemia children after transplantation compared to chronic
Suzanne Verlhac1, Flaviu Gabor2, Catherine Paillard3
1Referral Center for Sickle Cell Disease, Intercommunal Créteil Hospital, Robert-Debré Hospital APHP Paris, University Paris-Diderot, Paris, France.
Insights
Stem cell transplantation (SCT) significantly improved cerebral stenosis outcomes in children with sickle cell anaemia (SCA) compared to standard care. SCT prevented new stenosis and reduced existing cases over three years.
Area of Science:
- Hematology
- Neurology
- Pediatrics
Background:
- Sickle cell anaemia (SCA) is associated with an increased risk of cerebrovascular complications, including stenosis.
- Abnormal transcranial Doppler (TCD) findings in children with SCA indicate a higher risk of stroke.
- The DREPAGREFFE trial investigated the efficacy of stem cell transplantation (SCT) versus standard care in managing these risks.
Purpose of the Study:
- To evaluate the 3-year stenosis outcomes in stroke-free children with SCA and abnormal TCD.
- To compare the effectiveness of SCT against standard care (minimum 1-year chronic transfusion) in preventing and resolving cerebral stenosis.
Main Methods:
- The study enrolled 60 stroke-free children with SCA and abnormal TCD.
- Patients were randomized to either SCT (28 patients) or standard care (32 patients).
- Cerebral/cervical 3D time-of-flight magnetic resonance angiography was used to assess stenosis scores at enrollment and 3 years.
Main Results:
- At enrollment, 14 patients had stenosis; by 3 years, only 5 SCT patients versus 10 standard-care patients retained stenosis.
- Stenosis scores remained stable in the standard-care group but significantly improved in the SCT group (P=0.006).
- No new stenosis cases developed post-SCT, whereas two occurred in the standard-care group.
Conclusions:
- Stem cell transplantation demonstrates superior efficacy in improving and preventing cerebral stenosis in children with SCA compared to standard care.
- SCT offers a significant benefit for managing cerebrovascular complications in pediatric SCA patients.
- These findings support SCT as a potentially transformative treatment for reducing stroke risk in SCA.
Abstract:
We report here the 3-year stenosis outcome in 60 stroke-free children with sickle cell anaemia (SCA) and an abnormal transcranial Doppler history, enrolled in the DREPAGREFFE trial, which compared stem cell transplantation (SCT) with standard-care (chronic transfusion for 1-year minimum). Twenty-eight patients with matched sibling donors were transplanted, while 32 remained on standard-care. Stenosis scores were calculated after performing cerebral/cervical 3D time-of-flight magnetic resonance angiography. Fourteen patients had stenosis at enrollment, but only five SCT versus 10 standard-care patients still had stenosis at 3 years. Stenosis scores remained stable on standard-care, but significantly improved after SCT (P = 0·006). No patient developed stenosis after SCT, while two on standard-care did, indicating better stenosis prevention and improved outcome after SCT.
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