title: Haploidentical transplantation in children with unmanipulated peripheral blood stem cell graft: The need to look beyond post-transplantation cyclophosphamide in younger children.
abstract: Haploidentical transplantation with PTCY following marrow or PBSC graft has been associated with low incidence of GVHD in adults with similar data lacking in children. We report on the outcome of 25 patients <20 yr of age (median age 12 yr), undergoing a haploidentical PBSC transplantation for both malignant and non-malignant disorders. Engraftment was prompt and sustained. Cumulative incidences of acute GVHD and chronic GVHD were 40.3% and 16.7%, respectively. On subgroup analysis, it was evident that acute GVHD developed in 80% of patients <10 yr compared to only 13.3% in those between 10 and 20 yr [log-rank p = 0.001], despite similar graft composition with significantly higher NRM (60% vs. 0%; p = 0.001). The FFS was 63.5%; (79% in >10 yr and 40% in <10 yr, p = 0.01). Our data suggest that PTCY-based haploidentical PBSC transplantation is feasible in older children, but results in early and severe alloreactivity in younger children. These findings, despite being counterintuitive, could be explained by the variable metabolism of CY and oral mycophenolate in younger children indicating that PTCY-based approach as used in adults might not be adequate for younger children.
authors.affiliation: Department of Blood and Marrow Transplantation, Dharamshila Hospital and Research Centre, New Delhi, India.|Manashi Chakrabarti Foundation, Kolkata, India.
authors.forename: Sarita Rani
authors.identifier: 0000-0002-9988-3514
authors.initials: SR
authors.lastname: Jaiswal
authors.affiliation: Manashi Chakrabarti Foundation, Kolkata, India.
authors.forename: Aditi
authors.initials: A
authors.lastname: Chakrabarti
authors.affiliation: Manashi Chakrabarti Foundation, Kolkata, India.|Apollo Gleneagles Hospital, Kolkata, India.
authors.forename: Sumita
authors.initials: S
authors.lastname: Chatterjee
authors.affiliation: CSIR-Indian Institute of Chemical Biology, Kolkata, India.|Academy of Scientific and Innovative Research, CSIR-CRRI, New Delhi, India.
authors.forename: Kunal
authors.initials: K
authors.lastname: Ray
authors.affiliation: Department of Blood and Marrow Transplantation, Dharamshila Hospital and Research Centre, New Delhi, India.|Manashi Chakrabarti Foundation, Kolkata, India.
authors.forename: Suparno
authors.initials: S
authors.lastname: Chakrabarti
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D003520:Cyclophosphamide / Q000627:therapeutic use; D046148:Donor Selection; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000235:genetics / Q000517:prevention & control*; D006239:Haplotypes; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D015994:Incidence; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D010865:Pilot Projects; D014019:Tissue Donors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide
keywords: graft-versus-host disease; haploidentical; pediatric; peripheral blood stem cell; post-transplantation cyclophosphamide