Evidence map›Paper›PMID 33377129›Full record

ArticleCell reports. Medicine2020

ACCELERATE: A Patient-Powered Natural History Study Design Enabling Clinical and Therapeutic Discoveries in a Rare Disorder.

Sheila K Pierson, Johnson S Khor, Jasira Ziglar, Amy Liu, Katherine Floess, Erin NaPier, Alexander M Gorzewski, Mark-Avery Tamakloe, Victoria Powers, Faizaan Akhter and 24 more

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Cell reports. Medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02817997 (ACCELERATE), which is not on this map. Cited by 23 papers.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed
1.6field-weighted citation impact, top 14% 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.

NCT02817997 recruitingnot on this map

ACCELERATE (Advancing Castleman Care With an Electronic Longitudinal Registry, E-Repository, And Treatment/Effectiveness Research): An International Registry for Patients With Castleman Disease

Typeobservational_patient_registrySponsorUniversity of PennsylvaniaRan2016 to 2027Enrolled1,000ConditionsCastleman Disease, Castleman's Disease, Giant Lymph Node Hyperplasia, Angiofollicular Lymph Hyperplasia
3 · Its place in the literature

Who cites it

23 citing papers in PubMed, 32 citations in OpenAlex.

  1. Breast-implant associated unicentric castleman disease: a case report.Current problems in cancer. Case reports · 2026
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  17. How we manage idiopathic multicentric Castleman disease.Clinical advances in hematology & oncology : H&O · 2022
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  20. International definition of iMCD-TAFRO: future perspectives.Journal of clinical and experimental hematopathology : JCEH · 2022
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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

34 authors at 16 institutions in 7 countries.

Sheila K PiersonDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Johnson S KhorDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Jasira ZiglarDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Amy LiuDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Katherine FloessDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Erin NaPierDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Alexander M GorzewskiDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Mark-Avery TamakloeDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Victoria PowersDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Faizaan AkhterDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Eric HaljasmaaDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Raj JayanthanCastleman Disease Collaborative Network, Philadelphia, PA 19104, USA.
Arthur RubensteinDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Mileva RepaskyCastleman Disease Collaborative Network, Philadelphia, PA 19104, USA.
Kojo Elenitoba-JohnsonDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Jason RuthCastleman Disease Collaborative Network, Philadelphia, PA 19104, USA.
Bette JacobsCastleman Disease Collaborative Network, Philadelphia, PA 19104, USA.
Matthew StreetlyMyeloma and Plasma Cell Disorders, King's College London, London SE1 9RT, UK.
Linus AngenendtDepartment of Medicine A, University Hospital Münster, Münster 48149, Germany.
Jose Luis PatierServicio de Medicina Interna, Hospital Universitario Ramón y Cajal, Madrid 28034, Spain.
Simone FerreroDipartimento di Biotecnologie Molecolari e Scienze per la Salute, Università degli Studi di Torino, via Genova 3, Torino 10126, Italy.
Pier Luigi ZinzaniIRCCS Azienda Ospedaliero-Universitaria di Bologna, Istituto di Ematologia "Seràgnoli," Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale Università degli Studi, Bologna 40138, Italy.
Louis TerriouService de Médecine Interne, Institute for Translational Research in Inflammation University of Lille, Inserm, CHU Lille, 59000 Lille, France.
Corey CasperInfectious Disease Research Institute, Seattle, WA 98102, USA.
Elaine JaffeNational Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Christian HoffmannICH Study Center, Infektionsmedizinisches Centrum Hamburg, Hamburg 20095, Germany.
Eric OksenhendlerDepartment of Clinical Immunology, Hôpital Saint-Louis, 75010 Paris, France.
Alexander FossåDepartment of Oncology, Oslo University Hospital, 0188 Oslo, Norway.
Gordan SrkalovicClinical Trials Department, Sparrow Herbert-Herman Cancer Center, Lansing, MI 48912, USA.
Amy ChadburnDepartment of Pathology and Laboratory Medicine, Weill Cornell Medicine, New York, NY 10065, USA.
Thomas S UldrickVaccine and Infectious Diseases Division, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.
Megan LimDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Frits van RheeMyeloma Center, Winthrop P. Rockefeller Cancer Institute, University of Arkansas for Medical Sciences, Little Rock, AR 72205, USA.
David C FajgenbaumDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
University of Pennsylvania · USCornell University · USFred Hutch Cancer Center · USHôpital Saint-Louis · FRHospital Universitario Ramón y Cajal · ESInfectious Disease Research Institute · USInfektionsmedizinisches Centrum Hamburg · DEInserm · FRIstituto di Ematologia di Bologna · ITKing's College London · GBNational Institutes of Health · USOslo University Hospital · NOSparrow Hospital · USUniversity Hospital Münster · DEUniversity of Turin · ITWinthrop Rockefeller Foundation · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Geographically dispersed patients, inconsistent treatment tracking, and limited infrastructure slow research for many orphan diseases. We assess the feasibility of a patient-powered study design to overcome these challenges for Castleman disease, a rare hematologic disorder. Here, we report initial results from the ACCELERATE natural history registry. ACCELERATE includes a traditional physician-reported arm and a patient-powered arm, which enables patients to directly contribute medical data and biospecimens. This study design enables successful enrollment, with the 5-year minimum enrollment goal being met in 2 years. A median of 683 clinical, laboratory, and imaging data elements are captured per patient in the patient-powered arm compared with 37 in the physician-reported arm. These data reveal subgrouping characteristics, identify off-label treatments, support treatment guidelines, and are used in 17 clinical and translational studies. This feasibility study demonstrates that the direct-to-patient design is effective for collecting natural history data and biospecimens, tracking therapies, and providing critical research infrastructure.

Indexed as

Data CollectionResearch DesignAdolescentAdultAgedAged, 80 and overCastleman DiseaseChildChild, PreschoolFemaleHumansInfantMaleMiddle AgedRare DiseasesRegistriesCastleman diseasedirect-to-patientnatural history registryorphan diseasepatient-powered

Identifiers

PMID33377129
PMCPMC7762771
OpenAlexW3114598105

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.