Clinical Benefit of Response in Chronic Graft-versus-Host Disease

Yoshihiro Inamoto, Paul J. Martin, Xiaoyu Chai, Madan Jagasia, Jeanne Palmer, Joseph Pidala, Corey Cutler, Steven Z. Pavletic, Mukta Arora, David Jacobsohn, Paul A. Carpenter, Mary E D Flowers, Nandita D Khera, Georgia B. Vogelsang, Daniel Weisdorf, Barry E. Storer, Stephanie J. Lee

Research output: Contribution to journalArticle

32 Citations (Scopus)

Abstract

To determine whether changes in objective response measures proposed by the National Institutes of Health correlate with clinical benefit, such as symptom burden, quality of life, and survival outcomes, we analyzed data from a multicenter prospective cohort of 283 patients with chronic graft-versus-host disease requiring systemic treatment. The median follow-up time of survivors was 25.1 months (range, 5.4-47.7 months) after enrollment. Symptom measures included the Lee symptom scale and 10-point patient-reported symptoms. Quality-of-life measures included the Short Form-36, Functional Assessment of Cancer Therapy-Bone Marrow Transplantation, and Human Activities Profile. Overall and organ-specific responses were calculated by comparing manifestations at the 6-month visit and those at the enrollment visit using a provisional algorithm. Complete or partial responses were considered "response," and stable or progressive disease was considered "no response." Overall response rate at 6 months was 32%. Organ-specific response rates were 45% for skin, 23% for eyes, 32% for mouth, and 51% for gastrointestinal tract. Response at 6 months, as calculated according to the provisional response algorithm, was correlated with changes in symptom burden in patients with newly diagnosed chronic graft-versus-host disease, but not with changes in quality of life or survival outcomes. Modification of the algorithm or validation of other more meaningful clinical endpoints is warranted for future clinical trials of treatment for chronic graft-versus-host disease.

Original languageEnglish (US)
Pages (from-to)1517-1524
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Volume18
Issue number10
DOIs
StatePublished - Oct 2012
Externally publishedYes

Fingerprint

Graft vs Host Disease
Quality of Life
Bone Neoplasms
Survival
National Institutes of Health (U.S.)
Bone Marrow Transplantation
Human Activities
Survivors
Mouth
Gastrointestinal Tract
Therapeutics
Clinical Trials
Skin

Keywords

  • Allogeneic
  • Hematopoietic cell transplantation
  • National Institutes of Health
  • Response criteria

ASJC Scopus subject areas

  • Transplantation
  • Hematology

Cite this

Inamoto, Y., Martin, P. J., Chai, X., Jagasia, M., Palmer, J., Pidala, J., ... Lee, S. J. (2012). Clinical Benefit of Response in Chronic Graft-versus-Host Disease. Biology of Blood and Marrow Transplantation, 18(10), 1517-1524. https://doi.org/10.1016/j.bbmt.2012.05.016

Clinical Benefit of Response in Chronic Graft-versus-Host Disease. / Inamoto, Yoshihiro; Martin, Paul J.; Chai, Xiaoyu; Jagasia, Madan; Palmer, Jeanne; Pidala, Joseph; Cutler, Corey; Pavletic, Steven Z.; Arora, Mukta; Jacobsohn, David; Carpenter, Paul A.; Flowers, Mary E D; Khera, Nandita D; Vogelsang, Georgia B.; Weisdorf, Daniel; Storer, Barry E.; Lee, Stephanie J.

In: Biology of Blood and Marrow Transplantation, Vol. 18, No. 10, 10.2012, p. 1517-1524.

Research output: Contribution to journalArticle

Inamoto, Y, Martin, PJ, Chai, X, Jagasia, M, Palmer, J, Pidala, J, Cutler, C, Pavletic, SZ, Arora, M, Jacobsohn, D, Carpenter, PA, Flowers, MED, Khera, ND, Vogelsang, GB, Weisdorf, D, Storer, BE & Lee, SJ 2012, 'Clinical Benefit of Response in Chronic Graft-versus-Host Disease', Biology of Blood and Marrow Transplantation, vol. 18, no. 10, pp. 1517-1524. https://doi.org/10.1016/j.bbmt.2012.05.016
Inamoto, Yoshihiro ; Martin, Paul J. ; Chai, Xiaoyu ; Jagasia, Madan ; Palmer, Jeanne ; Pidala, Joseph ; Cutler, Corey ; Pavletic, Steven Z. ; Arora, Mukta ; Jacobsohn, David ; Carpenter, Paul A. ; Flowers, Mary E D ; Khera, Nandita D ; Vogelsang, Georgia B. ; Weisdorf, Daniel ; Storer, Barry E. ; Lee, Stephanie J. / Clinical Benefit of Response in Chronic Graft-versus-Host Disease. In: Biology of Blood and Marrow Transplantation. 2012 ; Vol. 18, No. 10. pp. 1517-1524.
@article{3932b687c09940c5998507f0c38d90fa,
title = "Clinical Benefit of Response in Chronic Graft-versus-Host Disease",
abstract = "To determine whether changes in objective response measures proposed by the National Institutes of Health correlate with clinical benefit, such as symptom burden, quality of life, and survival outcomes, we analyzed data from a multicenter prospective cohort of 283 patients with chronic graft-versus-host disease requiring systemic treatment. The median follow-up time of survivors was 25.1 months (range, 5.4-47.7 months) after enrollment. Symptom measures included the Lee symptom scale and 10-point patient-reported symptoms. Quality-of-life measures included the Short Form-36, Functional Assessment of Cancer Therapy-Bone Marrow Transplantation, and Human Activities Profile. Overall and organ-specific responses were calculated by comparing manifestations at the 6-month visit and those at the enrollment visit using a provisional algorithm. Complete or partial responses were considered {"}response,{"} and stable or progressive disease was considered {"}no response.{"} Overall response rate at 6 months was 32{\%}. Organ-specific response rates were 45{\%} for skin, 23{\%} for eyes, 32{\%} for mouth, and 51{\%} for gastrointestinal tract. Response at 6 months, as calculated according to the provisional response algorithm, was correlated with changes in symptom burden in patients with newly diagnosed chronic graft-versus-host disease, but not with changes in quality of life or survival outcomes. Modification of the algorithm or validation of other more meaningful clinical endpoints is warranted for future clinical trials of treatment for chronic graft-versus-host disease.",
keywords = "Allogeneic, Hematopoietic cell transplantation, National Institutes of Health, Response criteria",
author = "Yoshihiro Inamoto and Martin, {Paul J.} and Xiaoyu Chai and Madan Jagasia and Jeanne Palmer and Joseph Pidala and Corey Cutler and Pavletic, {Steven Z.} and Mukta Arora and David Jacobsohn and Carpenter, {Paul A.} and Flowers, {Mary E D} and Khera, {Nandita D} and Vogelsang, {Georgia B.} and Daniel Weisdorf and Storer, {Barry E.} and Lee, {Stephanie J.}",
year = "2012",
month = "10",
doi = "10.1016/j.bbmt.2012.05.016",
language = "English (US)",
volume = "18",
pages = "1517--1524",
journal = "Biology of Blood and Marrow Transplantation",
issn = "1083-8791",
publisher = "Elsevier Inc.",
number = "10",

}

TY - JOUR

T1 - Clinical Benefit of Response in Chronic Graft-versus-Host Disease

AU - Inamoto, Yoshihiro

AU - Martin, Paul J.

AU - Chai, Xiaoyu

AU - Jagasia, Madan

AU - Palmer, Jeanne

AU - Pidala, Joseph

AU - Cutler, Corey

AU - Pavletic, Steven Z.

AU - Arora, Mukta

AU - Jacobsohn, David

AU - Carpenter, Paul A.

AU - Flowers, Mary E D

AU - Khera, Nandita D

AU - Vogelsang, Georgia B.

AU - Weisdorf, Daniel

AU - Storer, Barry E.

AU - Lee, Stephanie J.

PY - 2012/10

Y1 - 2012/10

N2 - To determine whether changes in objective response measures proposed by the National Institutes of Health correlate with clinical benefit, such as symptom burden, quality of life, and survival outcomes, we analyzed data from a multicenter prospective cohort of 283 patients with chronic graft-versus-host disease requiring systemic treatment. The median follow-up time of survivors was 25.1 months (range, 5.4-47.7 months) after enrollment. Symptom measures included the Lee symptom scale and 10-point patient-reported symptoms. Quality-of-life measures included the Short Form-36, Functional Assessment of Cancer Therapy-Bone Marrow Transplantation, and Human Activities Profile. Overall and organ-specific responses were calculated by comparing manifestations at the 6-month visit and those at the enrollment visit using a provisional algorithm. Complete or partial responses were considered "response," and stable or progressive disease was considered "no response." Overall response rate at 6 months was 32%. Organ-specific response rates were 45% for skin, 23% for eyes, 32% for mouth, and 51% for gastrointestinal tract. Response at 6 months, as calculated according to the provisional response algorithm, was correlated with changes in symptom burden in patients with newly diagnosed chronic graft-versus-host disease, but not with changes in quality of life or survival outcomes. Modification of the algorithm or validation of other more meaningful clinical endpoints is warranted for future clinical trials of treatment for chronic graft-versus-host disease.

AB - To determine whether changes in objective response measures proposed by the National Institutes of Health correlate with clinical benefit, such as symptom burden, quality of life, and survival outcomes, we analyzed data from a multicenter prospective cohort of 283 patients with chronic graft-versus-host disease requiring systemic treatment. The median follow-up time of survivors was 25.1 months (range, 5.4-47.7 months) after enrollment. Symptom measures included the Lee symptom scale and 10-point patient-reported symptoms. Quality-of-life measures included the Short Form-36, Functional Assessment of Cancer Therapy-Bone Marrow Transplantation, and Human Activities Profile. Overall and organ-specific responses were calculated by comparing manifestations at the 6-month visit and those at the enrollment visit using a provisional algorithm. Complete or partial responses were considered "response," and stable or progressive disease was considered "no response." Overall response rate at 6 months was 32%. Organ-specific response rates were 45% for skin, 23% for eyes, 32% for mouth, and 51% for gastrointestinal tract. Response at 6 months, as calculated according to the provisional response algorithm, was correlated with changes in symptom burden in patients with newly diagnosed chronic graft-versus-host disease, but not with changes in quality of life or survival outcomes. Modification of the algorithm or validation of other more meaningful clinical endpoints is warranted for future clinical trials of treatment for chronic graft-versus-host disease.

KW - Allogeneic

KW - Hematopoietic cell transplantation

KW - National Institutes of Health

KW - Response criteria

UR - http://www.scopus.com/inward/record.url?scp=84866166246&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84866166246&partnerID=8YFLogxK

U2 - 10.1016/j.bbmt.2012.05.016

DO - 10.1016/j.bbmt.2012.05.016

M3 - Article

C2 - 22683612

AN - SCOPUS:84866166246

VL - 18

SP - 1517

EP - 1524

JO - Biology of Blood and Marrow Transplantation

JF - Biology of Blood and Marrow Transplantation

SN - 1083-8791

IS - 10

ER -