Evidence map›Paper›PMID 24844857›Full record

ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2014

Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria.

Jeremy Pantin, Xin Tian, Nancy Geller, Catalina Ramos, Lisa Cook, Elena Cho, Phillip Scheinberg, Sumithira Vasu, Hahn Khuu, David Stroncek and 4 more

Open access · hybridAbstract read
In one paragraph

Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
1.3field-weighted citation impact, top 20% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 27 citations in OpenAlex.

  1. Hematopoietic Cell Transplantation for Paroxysmal Nocturnal Hemoglobinuria in the Age of Eculizumab.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019
    Trial
  2. Allogeneic transplantation using CD34British journal of haematology · 2017
    Trial
  3. Article
  4. Review
  5. Article
  6. Article
  7. Article
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

14 authors at 2 institutions in 2 countries.

Jeremy PantinHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland; Division of Hematology and Oncology, Department of Medicine, Georgia Regents University, Augusta, Georgia.
Xin TianOffice of Biostatistics Research, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Nancy GellerOffice of Biostatistics Research, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Catalina RamosHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Lisa CookHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Elena ChoHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Phillip ScheinbergHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland; Hospital São José - Hospital Beneficência Portuguesa de São Paulo, São Paulo, Brazil.
Sumithira VasuHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland; Division of Hematology, Department of Medicine, Wexner Medical Center, Ohio State University, Columbus, Ohio.
Hahn KhuuDepartment of Transfusion Medicine, Clinical Research Center, National Institutes of Health, Bethesda, Maryland.
David StroncekDepartment of Transfusion Medicine, Clinical Research Center, National Institutes of Health, Bethesda, Maryland.
John BarrettHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Neal S YoungHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Theresa DonohueHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland.
Richard W ChildsHematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland. Electronic address: childsr@nih.gov.
National Institutes of Health · USNational Institutes of Health Clinical Center · US

Funding

Pathogenesis And Treatment Of Aplastic AnemiaZIAHL002315 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI YOUNG, NEAL S · 2009 to 2025
$60.1M
Allogeneic Immunotherapy for cancer and nonmalignant hematological disordersZIAHL002345 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI CHILDS, RICHARD · 2009 to 2025
$53.3M
Allogeneic Immunotherapy for cancer and nonmalignant hematological disordersZ01HL002345 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI CHILDS, RICHARD · 2000 to 2008
$6.5M
Development of novel cell and gene therapiesZIACL002120 · CLC · CLINICAL CENTER · PI STRONCEK, DAVID · 2009 to 2025
$0k
Intramural NIH HHS Z01 HL002345Intramural NIH HHS ZIA HL002345
6 · The paper itself

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis, venous thrombosis, and bone marrow failure. Seventeen patients with debilitating PNH, including 8 who were HLA-alloimmunized, underwent a reduced-intensity allogeneic hematopoietic cell transplantation (HCT). All received cyclophosphamide/fludarabine +/- antithymocyte globulin followed by a granulocyte colony-stimulating factor-mobilized HCT from an HLA-matched relative. Glycosylphosphatidylinositol-negative neutrophils were detectable after engraftment but disappeared completely at a median 100 days after transplantation. With a median follow-up of nearly 6 years, 15 patients (87.8%) survived, all without any evidence of PNH, transfusion independent, and off anticoagulation. Allogeneic reduced-intensity HCT remains a curative therapeutic option for PNH patients who are not candidates for eculizumab treatment.

Indexed as

AdultFemaleHematopoietic Stem Cell TransplantationHemoglobinuria, ParoxysmalHumansMaleSurvival AnalysisTransplantation ConditioningTreatment OutcomeVidarabineYoung AdultfludarabineVidarabineFludarabineHematopoietic cell transplantationParoxysmal nocturnal hemoglobinuriaReduced-intensitySurvival

Identifiers

PMID24844857
PMCPMC4157568
OpenAlexW2048034725

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.