A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia

Sonali Chaudhury*, M. Ayas, Colleen Rosen, Madeline Ma, M. Viqaruddin, Suhag Parikh, Sandhya Kharbanda, K. Y. Chiang, Ann Haight, Monica Bhatia, Greg Guilcher, Alexis Thompson, Shalini Shenoy

*Corresponding author for this work

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

Allogeneic hematopoietic cell transplantation (HCT) is curative in patients with β-thalassemia major. However, most reports on HCT outcomes lack long-term follow-up data with the exception of single-center reports. An international multicenter retrospective data collection and analysis was conducted in 176 β-thalassemia patients who were 1 year or beyond after first HCT to evaluate follow-up methods and outcomes at 7 centers. Median age at HCT was 5.5 years (range, .6 to 18.5), and median follow-up was 7 years (range, 1 to 20). HCT was predominantly from HLA-matched related donors (91%) with bone marrow as stem cell source (91%) and myeloablative conditioning regimens (88%). Late mortality or persistent chronic graft-versus-host disease (GVHD) was rare (<2%). Graft rejection was reported in 23% (24% of these occurred beyond 1 year) post-HCT. Of 119 patients with donor chimerism results available for ≥4 years post-HCT, 50% had >95%, 22% had 50% to 95%, 7% had 20% to 50% and 25 (21%) had <20% donor chimerism. Organ dysfunction was identified in 10% pre-HCT and in 20% post-HCT even without complete clinical details on all patients. Hypogonadism and elevated creatinine for age were most commonly reported and significantly higher in recipients ≥ 7 years at the time of HCT (P = .007) and in those with pre-existing morbidity before HCT (P = .02). Outcomes were unaffected by pre-HCT ferritin or GVHD. Mean z scores for height and weight were low at baseline and remained low post-HCT (79%), confirming that growth impairment from disease lacked recovery post-HCT during this follow-up period. HCT for β-thalassemia has a high rate of cure and low mortality, especially in the young and from HLA-matched related donors. Half of the number of recipients live with mixed chimerism that requires continued follow-up because of a risk of late graft rejection (14%). Organ function after HCT when <7 years of age was generally preserved. Hypogonadism, renal dysfunction, and growth impairment that failed to correct were late complications identified most frequently in older transplant recipients. Systematic follow-up of individual organs such as lung and heart were inadequate but important. These data support the development of simple measures of uniformly tracking long-term HCT outcomes and organ functions in children and adolescents who undergo HCT for thalassemia, allowing for systematic identification and implementation of standardized surveillance strategies and interventions.

Original languageEnglish (US)
Pages (from-to)1695-1700
Number of pages6
JournalBiology of Blood and Marrow Transplantation
Volume23
Issue number10
DOIs
StatePublished - Oct 1 2017

Fingerprint

Thalassemia
Cell Transplantation
Chimerism
Hypogonadism
Tissue Donors
Graft vs Host Disease
Mortality
beta-Thalassemia
Graft Rejection
Ferritins
Growth

Keywords

  • Late effects
  • Thalassemia
  • Transplant

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Chaudhury, Sonali ; Ayas, M. ; Rosen, Colleen ; Ma, Madeline ; Viqaruddin, M. ; Parikh, Suhag ; Kharbanda, Sandhya ; Chiang, K. Y. ; Haight, Ann ; Bhatia, Monica ; Guilcher, Greg ; Thompson, Alexis ; Shenoy, Shalini. / A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia. In: Biology of Blood and Marrow Transplantation. 2017 ; Vol. 23, No. 10. pp. 1695-1700.
@article{002ac8d73ea84e4882c3d132fe896d0d,
title = "A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia",
abstract = "Allogeneic hematopoietic cell transplantation (HCT) is curative in patients with β-thalassemia major. However, most reports on HCT outcomes lack long-term follow-up data with the exception of single-center reports. An international multicenter retrospective data collection and analysis was conducted in 176 β-thalassemia patients who were 1 year or beyond after first HCT to evaluate follow-up methods and outcomes at 7 centers. Median age at HCT was 5.5 years (range, .6 to 18.5), and median follow-up was 7 years (range, 1 to 20). HCT was predominantly from HLA-matched related donors (91{\%}) with bone marrow as stem cell source (91{\%}) and myeloablative conditioning regimens (88{\%}). Late mortality or persistent chronic graft-versus-host disease (GVHD) was rare (<2{\%}). Graft rejection was reported in 23{\%} (24{\%} of these occurred beyond 1 year) post-HCT. Of 119 patients with donor chimerism results available for ≥4 years post-HCT, 50{\%} had >95{\%}, 22{\%} had 50{\%} to 95{\%}, 7{\%} had 20{\%} to 50{\%} and 25 (21{\%}) had <20{\%} donor chimerism. Organ dysfunction was identified in 10{\%} pre-HCT and in 20{\%} post-HCT even without complete clinical details on all patients. Hypogonadism and elevated creatinine for age were most commonly reported and significantly higher in recipients ≥ 7 years at the time of HCT (P = .007) and in those with pre-existing morbidity before HCT (P = .02). Outcomes were unaffected by pre-HCT ferritin or GVHD. Mean z scores for height and weight were low at baseline and remained low post-HCT (79{\%}), confirming that growth impairment from disease lacked recovery post-HCT during this follow-up period. HCT for β-thalassemia has a high rate of cure and low mortality, especially in the young and from HLA-matched related donors. Half of the number of recipients live with mixed chimerism that requires continued follow-up because of a risk of late graft rejection (14{\%}). Organ function after HCT when <7 years of age was generally preserved. Hypogonadism, renal dysfunction, and growth impairment that failed to correct were late complications identified most frequently in older transplant recipients. Systematic follow-up of individual organs such as lung and heart were inadequate but important. These data support the development of simple measures of uniformly tracking long-term HCT outcomes and organ functions in children and adolescents who undergo HCT for thalassemia, allowing for systematic identification and implementation of standardized surveillance strategies and interventions.",
keywords = "Late effects, Thalassemia, Transplant",
author = "Sonali Chaudhury and M. Ayas and Colleen Rosen and Madeline Ma and M. Viqaruddin and Suhag Parikh and Sandhya Kharbanda and Chiang, {K. Y.} and Ann Haight and Monica Bhatia and Greg Guilcher and Alexis Thompson and Shalini Shenoy",
year = "2017",
month = "10",
day = "1",
doi = "10.1016/j.bbmt.2017.06.004",
language = "English (US)",
volume = "23",
pages = "1695--1700",
journal = "Biology of Blood and Marrow Transplantation",
issn = "1083-8791",
publisher = "Elsevier Inc.",
number = "10",

}

Chaudhury, S, Ayas, M, Rosen, C, Ma, M, Viqaruddin, M, Parikh, S, Kharbanda, S, Chiang, KY, Haight, A, Bhatia, M, Guilcher, G, Thompson, A & Shenoy, S 2017, 'A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia', Biology of Blood and Marrow Transplantation, vol. 23, no. 10, pp. 1695-1700. https://doi.org/10.1016/j.bbmt.2017.06.004

A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia. / Chaudhury, Sonali; Ayas, M.; Rosen, Colleen; Ma, Madeline; Viqaruddin, M.; Parikh, Suhag; Kharbanda, Sandhya; Chiang, K. Y.; Haight, Ann; Bhatia, Monica; Guilcher, Greg; Thompson, Alexis; Shenoy, Shalini.

In: Biology of Blood and Marrow Transplantation, Vol. 23, No. 10, 01.10.2017, p. 1695-1700.

Research output: Contribution to journalArticle

TY - JOUR

T1 - A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia

AU - Chaudhury, Sonali

AU - Ayas, M.

AU - Rosen, Colleen

AU - Ma, Madeline

AU - Viqaruddin, M.

AU - Parikh, Suhag

AU - Kharbanda, Sandhya

AU - Chiang, K. Y.

AU - Haight, Ann

AU - Bhatia, Monica

AU - Guilcher, Greg

AU - Thompson, Alexis

AU - Shenoy, Shalini

PY - 2017/10/1

Y1 - 2017/10/1

N2 - Allogeneic hematopoietic cell transplantation (HCT) is curative in patients with β-thalassemia major. However, most reports on HCT outcomes lack long-term follow-up data with the exception of single-center reports. An international multicenter retrospective data collection and analysis was conducted in 176 β-thalassemia patients who were 1 year or beyond after first HCT to evaluate follow-up methods and outcomes at 7 centers. Median age at HCT was 5.5 years (range, .6 to 18.5), and median follow-up was 7 years (range, 1 to 20). HCT was predominantly from HLA-matched related donors (91%) with bone marrow as stem cell source (91%) and myeloablative conditioning regimens (88%). Late mortality or persistent chronic graft-versus-host disease (GVHD) was rare (<2%). Graft rejection was reported in 23% (24% of these occurred beyond 1 year) post-HCT. Of 119 patients with donor chimerism results available for ≥4 years post-HCT, 50% had >95%, 22% had 50% to 95%, 7% had 20% to 50% and 25 (21%) had <20% donor chimerism. Organ dysfunction was identified in 10% pre-HCT and in 20% post-HCT even without complete clinical details on all patients. Hypogonadism and elevated creatinine for age were most commonly reported and significantly higher in recipients ≥ 7 years at the time of HCT (P = .007) and in those with pre-existing morbidity before HCT (P = .02). Outcomes were unaffected by pre-HCT ferritin or GVHD. Mean z scores for height and weight were low at baseline and remained low post-HCT (79%), confirming that growth impairment from disease lacked recovery post-HCT during this follow-up period. HCT for β-thalassemia has a high rate of cure and low mortality, especially in the young and from HLA-matched related donors. Half of the number of recipients live with mixed chimerism that requires continued follow-up because of a risk of late graft rejection (14%). Organ function after HCT when <7 years of age was generally preserved. Hypogonadism, renal dysfunction, and growth impairment that failed to correct were late complications identified most frequently in older transplant recipients. Systematic follow-up of individual organs such as lung and heart were inadequate but important. These data support the development of simple measures of uniformly tracking long-term HCT outcomes and organ functions in children and adolescents who undergo HCT for thalassemia, allowing for systematic identification and implementation of standardized surveillance strategies and interventions.

AB - Allogeneic hematopoietic cell transplantation (HCT) is curative in patients with β-thalassemia major. However, most reports on HCT outcomes lack long-term follow-up data with the exception of single-center reports. An international multicenter retrospective data collection and analysis was conducted in 176 β-thalassemia patients who were 1 year or beyond after first HCT to evaluate follow-up methods and outcomes at 7 centers. Median age at HCT was 5.5 years (range, .6 to 18.5), and median follow-up was 7 years (range, 1 to 20). HCT was predominantly from HLA-matched related donors (91%) with bone marrow as stem cell source (91%) and myeloablative conditioning regimens (88%). Late mortality or persistent chronic graft-versus-host disease (GVHD) was rare (<2%). Graft rejection was reported in 23% (24% of these occurred beyond 1 year) post-HCT. Of 119 patients with donor chimerism results available for ≥4 years post-HCT, 50% had >95%, 22% had 50% to 95%, 7% had 20% to 50% and 25 (21%) had <20% donor chimerism. Organ dysfunction was identified in 10% pre-HCT and in 20% post-HCT even without complete clinical details on all patients. Hypogonadism and elevated creatinine for age were most commonly reported and significantly higher in recipients ≥ 7 years at the time of HCT (P = .007) and in those with pre-existing morbidity before HCT (P = .02). Outcomes were unaffected by pre-HCT ferritin or GVHD. Mean z scores for height and weight were low at baseline and remained low post-HCT (79%), confirming that growth impairment from disease lacked recovery post-HCT during this follow-up period. HCT for β-thalassemia has a high rate of cure and low mortality, especially in the young and from HLA-matched related donors. Half of the number of recipients live with mixed chimerism that requires continued follow-up because of a risk of late graft rejection (14%). Organ function after HCT when <7 years of age was generally preserved. Hypogonadism, renal dysfunction, and growth impairment that failed to correct were late complications identified most frequently in older transplant recipients. Systematic follow-up of individual organs such as lung and heart were inadequate but important. These data support the development of simple measures of uniformly tracking long-term HCT outcomes and organ functions in children and adolescents who undergo HCT for thalassemia, allowing for systematic identification and implementation of standardized surveillance strategies and interventions.

KW - Late effects

KW - Thalassemia

KW - Transplant

UR - http://www.scopus.com/inward/record.url?scp=85025817376&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85025817376&partnerID=8YFLogxK

U2 - 10.1016/j.bbmt.2017.06.004

DO - 10.1016/j.bbmt.2017.06.004

M3 - Article

VL - 23

SP - 1695

EP - 1700

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

IS - 10

ER -