298 Progression of Cerebral Vascular Disease Post Transplant in a Child with Prior Sickle Cell Disease

Track: Poster Abstracts
Wednesday, February 11, 2015, 6:45 PM-7:45 PM
Grand Hall CD (Manchester Grand Hyatt)
Renee C Gresh, DO , Pediatric Hematology/Oncology, Nemours/AI duPont Children's Hospital, Wilmington, DE
Corinna L Schultz, MD, MSHP , Pediatric Hematology/Oncology, Nemours/AI duPont Children's Hospital, Wilmington, DE
Vinay Kandula, MD , Radiology, Nemours/AI duPont Children's Hospital, Wilmington, DE
Joseph H Piatt Jr., MD, FAAP , Pediatric Neurosurgery, Nemours/AI duPont Children's Hospital, Wilmington, DE
Emi Caywood, MD , BBMT, A.I. duPont Hospital for Children of the Nemours Foundation, Wilmington, DE
Robin Miller, MD , Hematology/Oncology, AI DuPont Hospital for Children, Wilmington, DE
E. Anders Kolb, MD , BBMT, A I duPont Hospital for Children, Wilmington, DE
Presentation recording not available for download or distribution as requested by the presenting author.

Progression of Cerebral Vascular Disease Post Transplant in a Child with Prior Sickle Cell Disease

We present a case report of a child with sickle cell disease who underwent a matched sibling bone marrow transplant for cerebral vascular disease and associated moyamoya.  At 4 years of age the patient had his first abnormal transcranial doppler ultrasound with elevated peak systolic velocity (PSV) along the right and left middle cerebral artery (MCA).  Follow-up MRI revealed severe narrowing of the right-sided M1 and A1 segments of the MCA along with evidence of multiple prior ischemic events.  MRI at 5 years of age showed progressive and severe narrowing of the right MCA and the anterior segment of the right anterior cerebral artery (ACA) along with development of moyamoya.  Follow up MRI demonstrated near complete occlusion of the right MCA, narrowing of the right ACA, and decreased perfusion within the right anterior MCA territory.  Because of his severe and progressive cerebral vasculopathy requiring chronic transfusion therapy, the patient underwent bone marrow transplant as curative treatment for his sickle cell disease with the intent to halt progression of his sickle cell induced vasculopathy.  The patient underwent a 10/10 matched sibling donor transplant with Busulfan/Cyclophosphamide conditioning and methotrexate/cyclosporine GVHD prophylaxis.  Engraftment occurred with 100% donor chimerism.  Four months post-bone marrow transplant (BMT), MRI showed stabilization of the progressive narrowing of the right MCA and ACA.  One year post-BMT there was improved perfusion in the MCA distribution. On routine follow up 3 years post-transplant, MRI showed new and severe narrowing of the left A1 segment along with evidence of interval ischemia.  Since BMT, the patient has been well appearing and without any signs or symptoms of overt neurologic events.  His hemoglobin levels have been in the normal range (12.2-13.5 g/dL) and his Hemoglobin S percentage has been 37% (donor with sickle cell trait).  He is currently undergoing a preoperative evaluation for synangiosis surgery.   Just as chronic transfusion therapy with resulting reduction in the Hemoglobin S percentage has been shown to halt progression of cerebral disease and prevent recurrent stroke in patients with sickle cell disease, it was hoped that curative bone marrow transplant would do the same.  Though this patient had a transient improvement in his vascular disease, he has now had an unexpected and significant progression.  The role of sickle cell trait in his progression is unclear.  Though bone marrow transplantation may be curative therapy for sickle cell disease, it may not halt the progression in all patients, especially those with severe disease.

Disclosures:
Nothing To Disclose
See more of: Poster Session 1: Pediatric Disorders
See more of: Poster Abstracts
<< Previous Presentation | Next Presentation >>