title: Pediatric hematology providers on referral for transplant evaluation for sickle cell disease: a regional perspective.
abstract: Hematology referral for evaluation is a key step for hematopoietic stem cell transplantation for sickle cell disease (SCD). Pediatric SCD providers in the US Northeast (New York-Mid-Atlantic and New England regions) were surveyed anonymously for perspectives and practices regarding transplant referral and compared by whether they practiced at SCD transplant centers. Data were analyzed using the Fisher exact test, χ test, and logistic regression. Half of the respondents practiced primarily at transplant sites. Most (79%) were enthusiastic about transplant for SCD and 78% had recently referred ≥1 child for evaluation. Overall, 77% limited referral to certain sickle hemoglobinopathies and 44% preferred referral for β-thalassemia to SCD. Indications selected for referral resembled current transplant criteria, plus family request or poor response to therapy. Referral for children on chronic transfusions predicted enthusiasm and prior referral. Many (66%) referred children with multiple SCD complications, even without matched sibling donors, 37% with sibling donors despite limited disease. Practitioners at transplant centers more commonly accepted event-free survival rates of ≤90% (P=0.002). Northeastern providers expressed varying enthusiasm for referral for evaluation based on eligibility, donor availability, and acceptable risk, with modestly more interest from practitioners at transplant centers. Differing provider perspectives may affect patient referral for transplant consideration.
authors.affiliation: *Department of Pediatric Hematology/Oncology, Naval Medical Center Portsmouth, Portsmouth, VA †Pediatric Hematology, Oncology and Stem Cell Transplantation §Department of Biostatistics, Mailman School of Public Health, Columbia University Medical Center ‡Department of Pediatrics, Division of General Pediatrics, Albert Einstein College of Medicine, Montefiore Medical Center, New York, NY.
authors.forename: Bethany
authors.initials: B
authors.lastname: Mikles
authors.forename: Monica
authors.initials: M
authors.lastname: Bhatia
authors.forename: Suzette O
authors.initials: SO
authors.lastname: Oyeku
authors.forename: Zhezhen
authors.initials: Z
authors.lastname: Jin
authors.forename: Nancy S
authors.initials: NS
authors.lastname: Green
mesh_terms: D000328:Adult; D000755:Anemia, Sickle Cell / Q000628:therapy*; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D003430:Cross-Sectional Studies; D018572:Disease-Free Survival; D005260:Female; D019538:Health Care Surveys; D006405:Hematology / Q000706:statistics & numerical data*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008875:Middle Aged; D015997:Neonatal Screening; D009511:New England; D010372:Pediatrics / Q000706:statistics & numerical data*; D011364:Professional Practice / Q000706:statistics & numerical data*; D012017:Referral and Consultation / Q000706:statistics & numerical data*; D055815:Young Adult; D017086:beta-Thalassemia / Q000628:therapy