Evidence map›Paper›PMID 24911148›Full record

ArticleThe Journal of clinical investigation2014

Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment.

Mitchell E Horwitz, Nelson J Chao, David A Rizzieri, Gwynn D Long, Keith M Sullivan, Cristina Gasparetto, John P Chute, Ashley Morris, Carolyn McDonald, Barbara Waters-Pick and 13 more

Registry-linked trialAbstract readClinical Trial, Phase I
In one paragraph

Article in The Journal of clinical investigation, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01221857 (Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies), which is not on this map. Cited by 121 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
121citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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.

NCT01221857 phase1 / phase2completednot on this map

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies

TypeinterventionalSponsorGamida Cell ltdRan2010 to 2013Enrolled12ConditionsAcute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Non-Hodgkin's LymphomaArmsNiCord®
3 · Its place in the literature

Who cites it

121 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
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  7. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2019
    Trial
  8. Trial
  9. Factors Associated with Long-Term Risk of Relapse after Unrelated Cord Blood Transplantation in Children with Acute Lymphoblastic Leukemia in Remission.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2017
    Trial
  10. Ex Vivo Mesenchymal Precursor Cell-Expanded Cord Blood Transplantation after Reduced-Intensity Conditioning Regimens Improves Time to Neutrophil Recovery.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2017
    Trial
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  16. Cryopreservation and Thawing ofJournal of hematology · 2026
    Article
  17. Review
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  19. Article
  20. Review

61 more citing papers are in PubMed but not listed here.

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

23 authors.

Mitchell E Horwitz
Nelson J Chao
David A Rizzieri
Gwynn D Long
Keith M Sullivan
Cristina Gasparetto
John P Chute
Ashley Morris
Carolyn McDonald
Barbara Waters-Pick
Patrick Stiff
Steven Wease
Amnon Peled
David Snyder
Einat Galamidi Cohen
Hadas Shoham
Efrat Landau
Etty Friend
Iddo Peleg
Dorit Aschengrau
Dima Yackoubov
Joanne Kurtzberg
Tony Peled

Funding

Women's Cancer Research ProgramP30CA014236 · NCI · DUKE UNIVERSITY · PI DONNA NIEDZWIECKI · 1985 to 2026
$174.8M
THERAPEUTIC APPROACHES TO OVARIAN/BREAST CANCER--HIGH DOSE CHEMOTHERAPY AND ABMSP01CA047741 · NCI · DUKE UNIVERSITY · PI HASSELBLAD, VICTOR · 1990 to 2013
$19.5M
NCI NIH HHS P01 CA047741NCI NIH HHS P01-CA047741-19NCI NIH HHS P30 CA014236
6 · The paper itself

Abstract

backgroundDelayed hematopoietic recovery is a major drawback of umbilical cord blood (UCB) transplantation. Transplantation of ex vivo-expanded UCB shortens time to hematopoietic recovery, but long-term, robust engraftment by the expanded unit has yet to be demonstrated. We tested the hypothesis that a UCB-derived cell product consisting of stem cells expanded for 21 days in the presence of nicotinamide and a noncultured T cell fraction (NiCord) can accelerate hematopoietic recovery and provide long-term engraftment.

methodsIn a phase I trial, 11 adults with hematologic malignancies received myeloablative bone marrow conditioning followed by transplantation with NiCord and a second unmanipulated UCB unit. Safety, hematopoietic recovery, and donor engraftment were assessed and compared with historical controls.

resultsNo adverse events were attributable to the infusion of NiCord. Complete or partial neutrophil and T cell engraftment derived from NiCord was observed in 8 patients, and NiCord engraftment remained stable in all patients, with a median follow-up of 21 months. Two patients achieved long-term engraftment with the unmanipulated unit. Patients transplanted with NiCord achieved earlier median neutrophil recovery (13 vs. 25 days, P < 0.001) compared with that seen in historical controls. The 1-year overall and progression-free survival rates were 82% and 73%, respectively.

conclusionUCB-derived hematopoietic stem and progenitor cells expanded in the presence of nicotinamide and transplanted with a T cell-containing fraction contain both short-term and long-term repopulating cells. The results justify further study of NiCord transplantation as a single UCB graft. If long-term safety is confirmed, NiCord has the potential to broaden accessibility and reduce the toxicity of UCB transplantation.

trial registrationClinicaltrials.gov NCT01221857.

fundingGamida Cell Ltd.

Indexed as

AdultCord Blood Stem Cell TransplantationFetal BloodGraft SurvivalHematologic NeoplasmsHematopoiesisHumansMiddle AgedNiacinamideTransplantation ChimeraTransplantation ConditioningTreatment OutcomeYoung AdultNiacinamide

Identifiers

PMID24911148
PMCPMC4071379

What OpenQuestion holds

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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.