BACKGROUND: Bone marrow transplantation (BMT) using human leukocyte antigen (HLA)-matched sibling donors can be curative for children with sickle cell anemia (SCA). However, minimal data exist regarding availability of HLA-identical matched siblings for transplant-eligible children, and family interest in pursuing transplantation. METHODS: We retrospectively analyzed a pediatric SCA cohort receiving chronic transfusions between July 2004 and January 2011. Data were analyzed regarding the number of full siblings and half-siblings, availability, and family interest in HLA testing the full siblings, and interest in proceeding with HLAmatched transplantation. RESULTS: Among 113 patients, 46 (41%) had at least 1 full sibling and 40 (35%) had an unaffected full sibling who could serve as a BMT donor. The families of 23 of these patients (58%) agreed to HLA-type sibling, 8 of whom (35%) were matched. Transfusion indications for families agreeing to HLA typing included stroke (46%) abnormal TCD (29%), acute chest syndrome (21%), and other CNS reasons (4%). Common reasons to decline HLA typing or transplantation included fear of the process, toxicities of the procedure, and comfort with current quality of life on transfusions. Only 8 of 113 (7%) were eligible for matched BMT, and only 3 (3%) underwent HLA-matched transplantation. Two unmatched children received haploidentical transplantation. CONCLUSIONS: Most families of children with SCA on chronic transfusions choose to proceed with HLA typing. However, when a matched sibling was identified, most families declined to proceed with matched-sibling transplantation. Discussing BMT as a treatment option, offering HLA typing and identifying barriers may improve acceptance of this treatment modality.
BACKGROUND: Bone marrow transplantation (BMT) using human leukocyte antigen (HLA)-matched sibling donors can be curative for children with sickle cell anemia (SCA). However, minimal data exist regarding availability of HLA-identical matched siblings for transplant-eligible children, and family interest in pursuing transplantation. METHODS: We retrospectively analyzed a pediatric SCA cohort receiving chronic transfusions between July 2004 and January 2011. Data were analyzed regarding the number of full siblings and half-siblings, availability, and family interest in HLA testing the full siblings, and interest in proceeding with HLAmatched transplantation. RESULTS: Among 113 patients, 46 (41%) had at least 1 full sibling and 40 (35%) had an unaffected full sibling who could serve as a BMT donor. The families of 23 of these patients (58%) agreed to HLA-type sibling, 8 of whom (35%) were matched. Transfusion indications for families agreeing to HLA typing included stroke (46%) abnormal TCD (29%), acute chest syndrome (21%), and other CNS reasons (4%). Common reasons to decline HLA typing or transplantation included fear of the process, toxicities of the procedure, and comfort with current quality of life on transfusions. Only 8 of 113 (7%) were eligible for matched BMT, and only 3 (3%) underwent HLA-matched transplantation. Two unmatched children received haploidentical transplantation. CONCLUSIONS: Most families of children with SCA on chronic transfusions choose to proceed with HLA typing. However, when a matched sibling was identified, most families declined to proceed with matched-sibling transplantation. Discussing BMT as a treatment option, offering HLA typing and identifying barriers may improve acceptance of this treatment modality.
Authors: Mari H Dallas; Brandon Triplett; David R Shook; Christine Hartford; Ashok Srinivasan; Joseph Laver; Russell Ware; Wing Leung Journal: Biol Blood Marrow Transplant Date: 2013-02-14 Impact factor: 5.742
Authors: Christian Brendel; Swaroopa Guda; Raffaele Renella; Daniel E Bauer; Matthew C Canver; Young-Jo Kim; Matthew M Heeney; Denise Klatt; Jonathan Fogel; Michael D Milsom; Stuart H Orkin; Richard I Gregory; David A Williams Journal: J Clin Invest Date: 2016-09-06 Impact factor: 14.808
Authors: Santosh L Saraf; Annie L Oh; Pritesh R Patel; Karen Sweiss; Matthew Koshy; Sally Campbell-Lee; Michel Gowhari; Shivi Jain; David Peace; John G Quigley; Irum Khan; Robert E Molokie; Nadim Mahmud; Victor R Gordeuk; Damiano Rondelli Journal: Biol Blood Marrow Transplant Date: 2018-04-12 Impact factor: 5.742
Authors: Lucía Gutiérrez; Michael J House; Nisha Vasavda; Emma Drašar; Isabel Gonzalez-Gascon Y Marin; Austin G Kulasekararaj; Tim G St Pierre; Swee L Thein Journal: PLoS One Date: 2015-09-25 Impact factor: 3.240