Evidence map›Paper›PMID 33351103›Full record

Trial reportBlood advances2020

Biomarker-guided preemption of steroid-refractory graft-versus-host disease with α-1-antitrypsin.

Stephanie C Gergoudis, Zachariah DeFilipp, Umut Özbek, Karamjeet S Sandhu, Aaron M Etra, Hannah K Choe, Carrie L Kitko, Francis Ayuk, Mina Aziz, Janna Baez and 19 more

Registry-linked trialOpen access · goldAbstract readClinical TrialMulticenter Study
In one paragraph

Trial report in Blood advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03459040 (A Proof of Concept Pilot Trial of Alpha-1-Antitrypsin for Pre-Emption Of Steroid-Refractory Acute GVHD), which is not on this map. Cited by 28 papers.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed
3.0field-weighted citation impact, top 7% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03459040 phase2completednot on this map

A Proof of Concept Pilot Trial of Alpha-1-Antitrypsin for Pre-Emption Of Steroid-Refractory Acute GVHD

TypeinterventionalSponsorJohn LevineRan2018 to 2020Enrolled30ConditionsGraft-versus-host-disease, GVHDArmsAlpha 1-Antitrypsin
3 · Its place in the literature

Who cites it

28 citing papers in PubMed, 36 citations in OpenAlex.

  1. Radiology. Imaging cancer · 2025
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  13. Planning GvHD preemptive therapy: risk factors, biomarkers, and prognostic scores.Hematology. American Society of Hematology. Education Program · 2023
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

29 authors at 13 institutions in 4 countries.

Stephanie C GergoudisTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Zachariah DeFilippBlood and Marrow Transplant Program, Massachusetts General Hospital, Boston, MA.
Umut ÖzbekBiostatistics Shared Resource Facility, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Karamjeet S SandhuDepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA.
Aaron M EtraTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Hannah K ChoeBlood and Marrow Transplantation Program, The Ohio State University Comprehensive Cancer Center, Columbus, OH.
Carrie L KitkoPediatric Blood and Marrow Transplantation Program, Vanderbilt University Medical Center, Nashville, TN.
Francis AyukDepartment of Stem Cell Transplantation, University Medical Center, Hamburg-Eppendorf, Germany.
Mina AzizTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Janna BaezTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Kaitlyn Ben-DavidTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Udomsak BunworasateDepartment of Medicine, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.
Isha GandhiTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Elizabeth O HexnerAbramson Cancer Center, University of Pennsylvania, Philadelphia, PA.
William J HoganBlood and Marrow Transplant Program, Division of Hematology, Mayo Clinic, Rochester, MN.
Ernst HollerDepartment of Hematology and Oncology, University Hospital Regensburg, Regensburg, Germany.
Stelios KasikisTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Steven M KowalykTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Jung-Yi LinBiostatistics Shared Resource Facility, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Pietro MerliDepartment of Pediatric Hematology/Oncology, Istituto di Ricovero e Cura a Carattere Scientifico Ospedale Pediatrico Bambino Gesù, Rome, Italy.
George MoralesTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Ryotaro NakamuraDepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA.
Ran ReshefBlood and Marrow Transplantation Program, Columbia University Irving Medical Center, New York, NY; and.
Wolf RöslerDepartment of Internal Medicine 5, Hematology/Oncology, University Hospital Erlangen, Erlangen, Germany.
Hrishikesh SrinageshTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Rachel YoungTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Yi-Bin ChenBlood and Marrow Transplant Program, Massachusetts General Hospital, Boston, MA.
James L M FerraraTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
John E LevineTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY.
Icahn School of Medicine at Mount Sinai · USCity Of Hope National Medical Center · USMassachusetts General Hospital · USBambino Gesù Children's Hospital · ITChulalongkorn University · THColumbia University Irving Medical Center · USMayo Clinic in Arizona · USThe Ohio State University · USUniversität Hamburg · DEUniversitätsklinikum Erlangen · DEUniversity Hospital Regensburg · DEUniversity of Pennsylvania · USVanderbilt University Medical Center · US

Funding

TUMOR VACCINES AND BONE MARROW TRANSPLANTATIONP01CA039542 · NCI · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI JAMES L. M. FERRARA · 1985 to 2026
$47.9M
THE TISCH CANCER INSTITUTE - CANCER CENTER SUPPORT GRANTP30CA196521 · NCI · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI Deanna L Benson · 2015 to 2026
$35.4M
NCI NIH HHS P01 CA039542NCI NIH HHS P30 CA196521
6 · The paper itself

Abstract

Steroid-refractory (SR) acute graft-versus-host disease (GVHD) remains a major cause of nonrelapse mortality (NRM) after allogeneic hematopoietic cell transplantation (HCT), but its occurrence is not accurately predicted by pre-HCT clinical risk factors. The Mount Sinai Acute GVHD International Consortium (MAGIC) algorithm probability (MAP) identifies patients who are at high risk for developing SR GVHD as early as 7 days after HCT based on the extent of intestinal crypt damage as measured by the concentrations of 2 serum biomarkers, suppressor of tumorigenesis 2 and regenerating islet-derived 3α. We conducted a multicenter proof-of-concept "preemptive" treatment trial of α-1-antitrypsin (AAT), a serine protease inhibitor with demonstrated activity against GVHD, in patients at high risk for developing SR GVHD. Patients were eligible if they possessed a high-risk MAP on day 7 after HCT or, if initially low risk, became high risk on repeat testing at day 14. Thirty high-risk patients were treated with twice-weekly infusions of AAT for a total of 16 doses, and their outcomes were compared with 90 high-risk near-contemporaneous MAGIC control patients. AAT treatment was well tolerated with few toxicities, but it did not lower the incidence of SR GVHD compared with controls (20% vs 14%, P = .56). We conclude that real-time biomarker-based risk assignment is feasible early after allogeneic HCT but that this dose and schedule of AAT did not change the incidence of SR acute GVHD. This trial was registered at www.clinicaltrials.gov as #NCT03459040.

Indexed as

Graft vs Host DiseaseHematopoietic Stem Cell TransplantationBiomarkersHumansSteroidsBiomarkersSteroids

Identifiers

PMID33351103
PMCPMC7756981
OpenAlexW3112351685

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.