Method
Data for this report come from the Florida Pediatric Bone Marrow
Transplant and Cell Therapy Consortium (FPBCC). The objectives of FPBCC
are to identify best pediatric HCT practices and improve survival of
children receiving HCT in Florida. FPBCC was founded in 2018 and
comprises 5 of the 6 pediatric transplant programs in the state of
Florida, USA. All FPBCC participating centers signed memoranda of
understanding and data use agreements and obtained institution-specific
IRB approvals for this retrospective data analysis. Data from
participating centers were downloaded from the enhanced data back to
center, a CIBMTR platform, which has access to a limited CIBMTR dataset.
Data were forwarded to the FPBCC statistical center, housed at the
University of Florida (Gainesville, FL, USA), where data were combined
into a single data set and analyzed. All participants signed an informed
consent for CIBMTR data collection. Transplant centers report detailed
data on consecutive hematopoietic cell transplantations to the
statistical center of CIBMTR, and compliance with this reporting is
monitored by on-site audits.
We describe demographics of pediatric HCT recipients, including gender,
age, race, as reported to the CIBMTR. Ethnic origin (e.g. Hispanic) was
not available in our dataset. Pre-transplant characteristics included
diagnosis, status of disease at transplant, time from diagnosis to
transplant, performance score, and number of pre-transplant
comorbidities. Transplant characteristics consist of type of donor, stem
cell source, Human Leukocyte Antigen (HLA) match, conditioning regimen,
and regimen intensity. Outcomes data include length of survival, cause
of death, and incidence and grade of GVHD. Myeloablative regimens were
defined as those using one or more of the following: total body
irradiation >500 cGy for a single dose or >800
cGy for fractionate, busulfan >7.2 mg/kg iv, melphalan
>150 mg/m2, and thiotepa ≥10 mg/kg.
Reduced intensity regimens were defined as those using melphalan ≤150
mg/m2, thiotepa <10 mg/kg, TBI
>200 cGy and ≤500 cGy as a single dose or ≤800 cGy
fractionated, busulfan ≤7.2 mg/kg. Non-myeloablative regimens used any
dose of ATG, fludarabine, cyclophosphamide, or TBI≤200 cGy. (8) Fully
HLA-matched unrelated BM or PB donors were matched at A, B, C, and DRB1
antigens by high resolution typing. For umbilical cord blood donors,
full match was defined as 6/6 HLA-antigen match (A, B, DRB1). A related
donor was considered haploidentical if ≥ 2 different antigens (A, B, C,
or DRB1) were mismatched. CIBMTR gathers and reports acute GVHD grade
following criteria published by Przepiorka et al. (9) Although currently
CIBMTR gathers data on individual organ involvement with cGVHD based on
NIH Consensus Criteria 2014; the data available through our platform
contained only information on extent of cGVHD as limited or extensive
based on definition by Shulman et al. (10) Limited cGVHD includes only
localized skin involvement and/or liver dysfunction, while any other
organ involvement is considered extensive.