Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes.
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Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes. / Marsh, Rebecca A; Rao, Kanchan; Satwani, Prakash; Lehmberg, Kai; Müller, Ingo; Li, Dandan; Kim, Mi-Ok; Fischer, Alain; Latour, Sylvain; Sedlacek, Petr; Barlogis, Vincent; Hamamoto, Kazuko; Kanegane, Hirokazu; Milanovich, Sam; Margolis, David A; Dimmock, David; Casper, James; Douglas, Dorothea N; Amrolia, Persis J; Veys, Paul; Kumar, Ashish R; Jordan, Michael B; Bleesing, Jack J; Filipovich, Alexandra H.
In: BLOOD, Vol. 121, No. 6, 6, 2013, p. 877-883.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes.
AU - Marsh, Rebecca A
AU - Rao, Kanchan
AU - Satwani, Prakash
AU - Lehmberg, Kai
AU - Müller, Ingo
AU - Li, Dandan
AU - Kim, Mi-Ok
AU - Fischer, Alain
AU - Latour, Sylvain
AU - Sedlacek, Petr
AU - Barlogis, Vincent
AU - Hamamoto, Kazuko
AU - Kanegane, Hirokazu
AU - Milanovich, Sam
AU - Margolis, David A
AU - Dimmock, David
AU - Casper, James
AU - Douglas, Dorothea N
AU - Amrolia, Persis J
AU - Veys, Paul
AU - Kumar, Ashish R
AU - Jordan, Michael B
AU - Bleesing, Jack J
AU - Filipovich, Alexandra H
PY - 2013
Y1 - 2013
N2 - There have been no studies on patient outcome after allogeneic hematopoietic cell transplantation (HCT) in patients with X-linked inhibitor of apoptosis (XIAP) deficiency. To estimate the success of HCT, we conducted an international survey of transplantation outcomes. Data were reported for 19 patients. Seven patients received busulfan-containing myeloablative conditioning (MAC) regimens. Eleven patients underwent reduced intensity conditioning (RIC) regimens predominantly consisting of alemtuzumab, fludarabine, and melphalan. One patient received an intermediate-intensity regimen. Survival was poor in the MAC group, with only 1 patient surviving (14%). Most deaths were from transplantation-related toxicities, including venoocclusive disease and pulmonary hemorrhage. Of the 11 patients who received RIC, 6 are currently surviving at a median of 570 days after HCT (55%). Preparative regimen and HLH activity affected outcomes, and of RIC patients reported to be in remission from HLH, survival is 86% (P = .03). We conclude that MAC regimens should not be used for patients with XIAP deficiency. It is possible that the loss of XIAP and its antiapoptotic functions contributes to the high incidence of toxicities observed with MAC regimens. RIC regimens should be pursued with caution and, if possible, efforts should be made to ensure HLH remission before HCT in these patients.
AB - There have been no studies on patient outcome after allogeneic hematopoietic cell transplantation (HCT) in patients with X-linked inhibitor of apoptosis (XIAP) deficiency. To estimate the success of HCT, we conducted an international survey of transplantation outcomes. Data were reported for 19 patients. Seven patients received busulfan-containing myeloablative conditioning (MAC) regimens. Eleven patients underwent reduced intensity conditioning (RIC) regimens predominantly consisting of alemtuzumab, fludarabine, and melphalan. One patient received an intermediate-intensity regimen. Survival was poor in the MAC group, with only 1 patient surviving (14%). Most deaths were from transplantation-related toxicities, including venoocclusive disease and pulmonary hemorrhage. Of the 11 patients who received RIC, 6 are currently surviving at a median of 570 days after HCT (55%). Preparative regimen and HLH activity affected outcomes, and of RIC patients reported to be in remission from HLH, survival is 86% (P = .03). We conclude that MAC regimens should not be used for patients with XIAP deficiency. It is possible that the loss of XIAP and its antiapoptotic functions contributes to the high incidence of toxicities observed with MAC regimens. RIC regimens should be pursued with caution and, if possible, efforts should be made to ensure HLH remission before HCT in these patients.
KW - Humans
KW - Adolescent
KW - Young Adult
KW - Europe
KW - Child
KW - Survival Analysis
KW - Survival Rate
KW - Combined Modality Therapy
KW - Child, Preschool
KW - Infant
KW - United States
KW - Retrospective Studies
KW - Mutation
KW - Remission Induction
KW - Transplantation, Homologous
KW - Japan
KW - Hemorrhage/etiology
KW - Antineoplastic Combined Chemotherapy Protocols/therapeutic use
KW - Outcome Assessment (Health Care)/statistics & numerical data
KW - Transplantation Conditioning/methods
KW - Genetic Diseases, X-Linked/genetics/mortality/therapy
KW - Hematopoietic Stem Cell Transplantation/adverse effects/methods
KW - Hepatic Veno-Occlusive Disease/etiology
KW - Lung/blood supply
KW - Lymphoproliferative Disorders/genetics/mortality/therapy
KW - X-Linked Inhibitor of Apoptosis Protein/genetics
KW - Humans
KW - Adolescent
KW - Young Adult
KW - Europe
KW - Child
KW - Survival Analysis
KW - Survival Rate
KW - Combined Modality Therapy
KW - Child, Preschool
KW - Infant
KW - United States
KW - Retrospective Studies
KW - Mutation
KW - Remission Induction
KW - Transplantation, Homologous
KW - Japan
KW - Hemorrhage/etiology
KW - Antineoplastic Combined Chemotherapy Protocols/therapeutic use
KW - Outcome Assessment (Health Care)/statistics & numerical data
KW - Transplantation Conditioning/methods
KW - Genetic Diseases, X-Linked/genetics/mortality/therapy
KW - Hematopoietic Stem Cell Transplantation/adverse effects/methods
KW - Hepatic Veno-Occlusive Disease/etiology
KW - Lung/blood supply
KW - Lymphoproliferative Disorders/genetics/mortality/therapy
KW - X-Linked Inhibitor of Apoptosis Protein/genetics
U2 - 10.1182/blood-2012-06-432500
DO - 10.1182/blood-2012-06-432500
M3 - SCORING: Journal article
C2 - 23131490
VL - 121
SP - 877
EP - 883
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 6
M1 - 6
ER -
