Pediatric Hematology Providers on Referral for Transplant Evaluation for Sickle Cell Disease

Abstract

Summary 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 anony-mously 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, χ2 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). North-eastern 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.

Decision making in pediatric hematopoietic cell transplantation: Influential factors vary among diseases

“…The urgency to undergo HCT for malignancy came from a belief that HCT is best . Those with nonmalignant diseases recognized HCT could be pursued at a later, more optimal time . The conflict and optimization of treatment in those considering HCT for nonmalignant diseases was sometimes influenced by disease severity and previous disease complications .…”

“…“Creating an awareness of timing” was dominant among physicians. Age, disease type, and severity of disease were factors considered in the timing of HCT in two of the three studies looking at physician perspective . Parents awareness of timing also stemmed from disease type and severity of disease, but also availability of a donor .…”

“…Alternatively, those pursing HCT for SCD or other nonmalignant diseases face conflict through recognizing options outside of HCT, and are yearning for a better quality of life. Stakeholders often appreciate disease severity and donor availability when weighing the decision of HCT in nonmalignant diseases . Much of the data on nonmalignant diseases represent SCD.…”

“…Stakeholders considering HCT for malignancy perceived HCT as the only option, 16,17,20,22 while those considering HCT for nonmalignant diseases perceived options other than HCT. 19,[24][25][26][27][28]32,34 Some stakeholders actually preferred supportive care strategies over HCT for SCD and MPS1. 11,19,25,34 Stakeholders considering HCT for malignancy felt there was no viable alternative outside of HCT, as death was the only alternative.…”

Paediatric haematologists’ attitudes regarding haematopoietic cell transplantation as treatment for sickle cell disease

“…Despite the high morbidity and mortality associated with SCD, haematopoietic cell transplantation (HCT), which is curative, remains underutilized (Center for International Blood & Marrow Transplant, ; Angelucci et al , ; Mikles et al , ; Bhatia & Sheth, ). For nearly 20 years, human leucocyte antigen (HLA) matched HCT in patients with SCD has demonstrated a 5‐year survival exceeding 90%, low rates of graft‐versus‐host disease and improved quality of life post‐transplantation (Shenoy, ; Bhatia & Walters, ; Hsieh et al , ; Fitzhugh et al , ; Bhatia et al , ; Gluckman et al , ).…”