Abstract
The Stroke Prevention Trial in Sickle Cell Anemia (STOP I) demonstrated a 90% risk reduction of first stroke in high-risk pediatric patients with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT) (
Sickle Cell Centers . | N = 25 . |
---|---|
TCD <170 cm/sec | |
Repeat every 6 mo | 4 |
Repeat yearly | 13 |
Repeat every 1–2 y | 4 |
Repeat every 2 y | 1 |
TCD 170–200 cm/sec | |
Repeat every mo | 7 |
Repeat every 2 mo | 3 |
Repeat every 3 mo | 6 |
Repeat every 4–5 mo | 3 |
Repeat every 6 mo | 6 |
TCD >200 cm/sec | |
Repeat every mo | 14 |
Repeat every 2 mo | 1 |
Repeat every 3 mo | 3 |
Sickle Cell Centers . | N = 25 . |
---|---|
TCD <170 cm/sec | |
Repeat every 6 mo | 4 |
Repeat yearly | 13 |
Repeat every 1–2 y | 4 |
Repeat every 2 y | 1 |
TCD 170–200 cm/sec | |
Repeat every mo | 7 |
Repeat every 2 mo | 3 |
Repeat every 3 mo | 6 |
Repeat every 4–5 mo | 3 |
Repeat every 6 mo | 6 |
TCD >200 cm/sec | |
Repeat every mo | 14 |
Repeat every 2 mo | 1 |
Repeat every 3 mo | 3 |
Based on clinical observations, participants estimated the percentage of patients with eventual worsening of TCD velocity to >200 cm/sec and the approximate number of months to reach that endpoint. Responses indicated an average of 23% and 11.3 months, respectively. Of the 25 centers, all but one indicated that patients with an abnormal TCD (>200 cm/sec) were typically placed on CTT. Four participants stated that results from the STOP trial were the basis for their decision. Fifteen centers indicated that the youngest age they would initiate CTT was 1–2 years. Only 2 directors would consider CTT in children <1 year, and 3 would not initiate CTT until age 5. Although the NHLBI guidelines suggest a hemoglobin S (Hb S) target of <30%, only 5 directors stated that this was their primary factor in determining frequency of CTT. In terms of typical CTT duration, 68% said therapy was indefinite or lifelong, 20% said <1 year, 4% said 2 years, and 4% said until age 18. Although the number of participants was limited, responses indicate there is a relatively wide variation in the application of the NHLBI recommendations for TCD use and CTT in pediatric patients with SCD. The importance of these findings should be correlated both with a more extensive sample of SCD centers and the reported incidence of stroke in the pediatric population.
Disclosures: Dr. Mulberg and Ms. Magia are employees of McNeil Pediatrics. Dr. Rigdon is an employee of Icagen, Inc. Dr. Behm was an employee of McNeil Pediatrics at the time of the survey, but is currently an employee of Merck Pharmaceuticals.; Dr. Adams is a consultant for Bristol-Myers Squibb, Sanofi-Aventis, Boehringer Ingelheim, Merck Pharmaceuticals, and the Veterans Affairs Department. Consulting agreements are limited to ad-hoc meetings and one time payments; no retainers are involved. Dr. Drachtman is a consultant for McNeil Pediatrics.; Dr. Mulberg owns stock in Johnson & Johnson. Dr. Rigdon owns stock in Icagen, Inc.; Dr. Adams’ research support is primarily from NHLBI-NIH.; Dr. Adams receives honoraria from Novartis.; Dr. Adams is on Advisory Groups for Boehringer Ingelheim, Bristol-Myers Squibb, and Sanofi-Aventis partnership in the area of stroke. Dr. Drachtman is a member of the Speakers Bureau for Sanofi-Aventis and Novartis.; Dr. Adams has given lectures in the last 2 years sponsored by Boehringer Ingelheim, Bristol-Myers Squibb, Sanofi-Aventis, and Novartis. Within the last 2 years, Dr. Adams has received small (<$3,000) unrestricted educational grants or support for training courses from Nicolet, Bristol-Myers Squibb, and Boehringer Ingelheim.
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