Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia
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Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia. / Strahm, Brigitte; Loewecke, Felicia; Niemeyer, Charlotte M; Albert, Michael; Ansari, Marc; Bader, Peter; Bertrand, Yves; Burkhardt, Birgit; Da Costa, Lydie M; Ferster, Alina; Fischer, Alexandra; Güngör, Tayfun; Gruhn, Bernd; Hainmann, Ina; Kapp, Friedrich; Lang, Peter; Müller, Ingo; Schulz, Ansgar; Szvetnik, Amina; Wlodarski, Marcin; Noellke, Peter; Leblanc, Thierry; Dalle, Jean-Hugues.
in: BLOOD ADV, Jahrgang 4, Nr. 8, 28.04.2020, S. 1760-1769.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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T1 - Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia
AU - Strahm, Brigitte
AU - Loewecke, Felicia
AU - Niemeyer, Charlotte M
AU - Albert, Michael
AU - Ansari, Marc
AU - Bader, Peter
AU - Bertrand, Yves
AU - Burkhardt, Birgit
AU - Da Costa, Lydie M
AU - Ferster, Alina
AU - Fischer, Alexandra
AU - Güngör, Tayfun
AU - Gruhn, Bernd
AU - Hainmann, Ina
AU - Kapp, Friedrich
AU - Lang, Peter
AU - Müller, Ingo
AU - Schulz, Ansgar
AU - Szvetnik, Amina
AU - Wlodarski, Marcin
AU - Noellke, Peter
AU - Leblanc, Thierry
AU - Dalle, Jean-Hugues
N1 - © 2020 by The American Society of Hematology.
PY - 2020/4/28
Y1 - 2020/4/28
N2 - Diamond-Blackfan anemia (DBA) is a congenital pure red cell aplasia associated with congenital abnormalities and cancer predisposition. Allogeneic hematopoietic stem cell transplantation (HSCT) can correct the hematological phenotype and is indicated in transfusion-dependent patients. In 70 children reported to the German DBA and French HSCT registries, HSCT was performed from 1985 to 2017. Median age at HSCT was 5.5 years (range, 0.9-17.3 years). Two-thirds of patients (64%) were transplanted from a matched sibling donor (MSD), and most procedures were performed after the year 1999 (73%). Primary engraftment was achieved in all patients. One patient developed secondary graft failure. Cumulative incidence of acute graft-versus-host disease (GVHD) was 24% for °II-IV (95% confidence interval [CI], 16% to 37%) and 7% for °III-IV (95% CI, 3% to 17%); cumulative incidence of chronic GVHD was 11% (95% CI, 5% to 22%). The probability of chronic GVHD-free survival (cGFS) was 87% (95% CI, 79% to 95%) and significantly improved over time (<2000: 68% [95% CI, 47% to 89%] vs ≥2000: 94% [95% CI, 87% to 100%], P < .01). cGFS was comparable following HSCT from a MSD and an unrelated donor (UD). Of note, no severe chronic GVHD or deaths were reported following MSD-HSCT after 1999. The difference of cGFS in children transplanted <10 years of age compared with older patients did not reach statistical significance (<10 years: 90% [95% CI, 81% to 99%] vs 10-18 years 78% [95% CI, 58% to 98%]). In summary, these data indicate that HSCT is efficient and safe in young DBA patients and should be considered if a MSD or matched UD is available. HSCT for transfusion dependency only must be critically discussed in older patients.
AB - Diamond-Blackfan anemia (DBA) is a congenital pure red cell aplasia associated with congenital abnormalities and cancer predisposition. Allogeneic hematopoietic stem cell transplantation (HSCT) can correct the hematological phenotype and is indicated in transfusion-dependent patients. In 70 children reported to the German DBA and French HSCT registries, HSCT was performed from 1985 to 2017. Median age at HSCT was 5.5 years (range, 0.9-17.3 years). Two-thirds of patients (64%) were transplanted from a matched sibling donor (MSD), and most procedures were performed after the year 1999 (73%). Primary engraftment was achieved in all patients. One patient developed secondary graft failure. Cumulative incidence of acute graft-versus-host disease (GVHD) was 24% for °II-IV (95% confidence interval [CI], 16% to 37%) and 7% for °III-IV (95% CI, 3% to 17%); cumulative incidence of chronic GVHD was 11% (95% CI, 5% to 22%). The probability of chronic GVHD-free survival (cGFS) was 87% (95% CI, 79% to 95%) and significantly improved over time (<2000: 68% [95% CI, 47% to 89%] vs ≥2000: 94% [95% CI, 87% to 100%], P < .01). cGFS was comparable following HSCT from a MSD and an unrelated donor (UD). Of note, no severe chronic GVHD or deaths were reported following MSD-HSCT after 1999. The difference of cGFS in children transplanted <10 years of age compared with older patients did not reach statistical significance (<10 years: 90% [95% CI, 81% to 99%] vs 10-18 years 78% [95% CI, 58% to 98%]). In summary, these data indicate that HSCT is efficient and safe in young DBA patients and should be considered if a MSD or matched UD is available. HSCT for transfusion dependency only must be critically discussed in older patients.
U2 - 10.1182/bloodadvances.2019001210
DO - 10.1182/bloodadvances.2019001210
M3 - SCORING: Journal article
C2 - 32343795
VL - 4
SP - 1760
EP - 1769
JO - BLOOD ADV
JF - BLOOD ADV
SN - 2473-9529
IS - 8
ER -
