Late Mortality after Allogeneic Bone Marrow Transplantation in Childhood for Bone Marrow Failure Syndromes and Severe Aplastic Anemia
(2019) In Biology of Blood and Marrow Transplantation 25(4). p.749-755- Abstract
Children with bone marrow failure syndromes and severe aplastic anemia (SAA) are treated with allogeneic blood or marrow transplantation (BMT). However, there is a paucity of studies examining late mortality risk after allogeneic BMT performed in childhood for bone marrow failure syndromes and SAA and evaluating how this risk differs between these diseases. We investigated cause-specific late mortality in 2-year survivors of allogeneic BMT for bone marrow failure syndromes and SAA performed before age 22 years between 1974 and 2010 at 2 US transplantation centers. Vital status information was collected from medical records, the National Death Index, and Accurint databases. Overall survival was calculated using Kaplan-Meier techniques.... (More)
Children with bone marrow failure syndromes and severe aplastic anemia (SAA) are treated with allogeneic blood or marrow transplantation (BMT). However, there is a paucity of studies examining late mortality risk after allogeneic BMT performed in childhood for bone marrow failure syndromes and SAA and evaluating how this risk differs between these diseases. We investigated cause-specific late mortality in 2-year survivors of allogeneic BMT for bone marrow failure syndromes and SAA performed before age 22 years between 1974 and 2010 at 2 US transplantation centers. Vital status information was collected from medical records, the National Death Index, and Accurint databases. Overall survival was calculated using Kaplan-Meier techniques. The standardized mortality ratio (SMR) was calculated using age- sex-, and calendar-specific mortality rates from the Centers for Disease Control and Prevention. Among the 2-year survivors of bone marrow failure syndromes (n = 120) and SAA (n = 147), there were 15 and 19 deaths, respectively, yielding an overall survival of 86.4% for bone marrow failure syndromes and 93.1% for SAA at 15 years post-BMT. Compared with the general population, patients with bone marrow failure syndromes were at a higher risk for premature death (SMR, 22.7; 95% CI, 13.1 to 36.2) compared with those with SAA (SMR, 4.5; 95% CI, 2.8 to 7.0) (P <.0001). The elevated relative risk persisted at ≥15 years after BMT for both diseases. The hazard of all-cause late mortality was 2.9-fold (95% CI, 1.1 to 7.3) higher in patients with bone marrow failure syndromes compared with those with SAA. The high late mortality risk in recipients of allogeneic BMT in childhood for bone marrow failure syndromes calls for intensified life-long follow-up.
(Less)
- author
- organization
- publishing date
- 2019
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- Allogeneic BMT, Bone marrow failure syndrome, Childhood, Late mortality, Severe aplastic anemia
- in
- Biology of Blood and Marrow Transplantation
- volume
- 25
- issue
- 4
- pages
- 749 - 755
- publisher
- Elsevier
- external identifiers
-
- scopus:85060719208
- pmid:30578940
- ISSN
- 1083-8791
- DOI
- 10.1016/j.bbmt.2018.12.063
- language
- English
- LU publication?
- yes
- id
- 5faa113c-164a-4df8-a43c-373e369ef6c3
- date added to LUP
- 2019-02-07 08:50:04
- date last changed
- 2024-02-14 17:23:32
@article{5faa113c-164a-4df8-a43c-373e369ef6c3,
abstract = {{<p>Children with bone marrow failure syndromes and severe aplastic anemia (SAA) are treated with allogeneic blood or marrow transplantation (BMT). However, there is a paucity of studies examining late mortality risk after allogeneic BMT performed in childhood for bone marrow failure syndromes and SAA and evaluating how this risk differs between these diseases. We investigated cause-specific late mortality in 2-year survivors of allogeneic BMT for bone marrow failure syndromes and SAA performed before age 22 years between 1974 and 2010 at 2 US transplantation centers. Vital status information was collected from medical records, the National Death Index, and Accurint databases. Overall survival was calculated using Kaplan-Meier techniques. The standardized mortality ratio (SMR) was calculated using age- sex-, and calendar-specific mortality rates from the Centers for Disease Control and Prevention. Among the 2-year survivors of bone marrow failure syndromes (n = 120) and SAA (n = 147), there were 15 and 19 deaths, respectively, yielding an overall survival of 86.4% for bone marrow failure syndromes and 93.1% for SAA at 15 years post-BMT. Compared with the general population, patients with bone marrow failure syndromes were at a higher risk for premature death (SMR, 22.7; 95% CI, 13.1 to 36.2) compared with those with SAA (SMR, 4.5; 95% CI, 2.8 to 7.0) (P <.0001). The elevated relative risk persisted at ≥15 years after BMT for both diseases. The hazard of all-cause late mortality was 2.9-fold (95% CI, 1.1 to 7.3) higher in patients with bone marrow failure syndromes compared with those with SAA. The high late mortality risk in recipients of allogeneic BMT in childhood for bone marrow failure syndromes calls for intensified life-long follow-up.</p>}},
author = {{Holmqvist, Anna Sällfors and Chen, Yanjun and Wu, Jessica and Battles, Kevin and Francisco, Liton and Hageman, Lindsey and Kung, Michelle and Ness, Emily and Parman, Mariel and Winther, Jeanette Falck and Rosenthal, Joseph and Arora, Mukta and Armenian, Saro H. and Bhatia, Smita}},
issn = {{1083-8791}},
keywords = {{Allogeneic BMT; Bone marrow failure syndrome; Childhood; Late mortality; Severe aplastic anemia}},
language = {{eng}},
number = {{4}},
pages = {{749--755}},
publisher = {{Elsevier}},
series = {{Biology of Blood and Marrow Transplantation}},
title = {{Late Mortality after Allogeneic Bone Marrow Transplantation in Childhood for Bone Marrow Failure Syndromes and Severe Aplastic Anemia}},
url = {{http://dx.doi.org/10.1016/j.bbmt.2018.12.063}},
doi = {{10.1016/j.bbmt.2018.12.063}},
volume = {{25}},
year = {{2019}},
}