Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.

dc.contributor.author

Mahadeo, Kris M

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Tewari, Priti

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Parikh, Suhag H

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Driscoll, Timothy A

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Page, Kristin

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Martin, Paul L

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Kurtzberg, Joanne

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Prasad, Vinod K

dc.date.accessioned

2022-03-23T19:20:39Z

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2022-03-23T19:20:39Z

dc.date.issued

2015-11

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2022-03-23T19:20:38Z

dc.description.abstract

The use of HSCT is the only potentially curative treatment for CAMT, but access is limited by the availability of suitable donors. We report five consecutive patients with CAMT who received MAC and partially HLA-mismatched, UCBT (unrelated, n = 4). Median times to neutrophil (>500/μL) and platelet (≥20 000 and ≥50 000/μL) engraftment were 19, 57, and 70 days, respectively. Acute GvHD, grade II, developed in one patient, who subsequently developed limited chronic GvHD. At median follow-up of 14 yr, all patients are alive with sustained donor cell engraftment. To our knowledge, this is the largest single-center series of UCBT for patients with this disease and suggests that UCBT is a successful curative option for patients with CAMT.

dc.identifier.issn

1397-3142

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1399-3046

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https://hdl.handle.net/10161/24646

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eng

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Wiley

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Pediatric transplantation

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10.1111/petr.12577

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Humans

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Thrombocytopenia

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Graft vs Host Disease

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Treatment Outcome

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Transplantation Conditioning

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Cord Blood Stem Cell Transplantation

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Follow-Up Studies

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Adolescent

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Child

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Child, Preschool

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Infant

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Infant, Newborn

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Female

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Male

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Congenital Bone Marrow Failure Syndromes

dc.title

Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation.

dc.type

Journal article

duke.contributor.orcid

Parikh, Suhag H|0000-0002-6066-9852

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Page, Kristin|0000-0001-9670-8828

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Martin, Paul L|0000-0001-8141-5678

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Kurtzberg, Joanne|0000-0002-3370-0703

pubs.begin-page

753

pubs.end-page

757

pubs.issue

7

pubs.organisational-group

Duke

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School of Medicine

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Faculty

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Clinical Science Departments

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Institutes and Centers

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Pathology

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Pediatrics

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Duke Cancer Institute

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Institutes and Provost's Academic Units

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Initiatives

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Duke Innovation & Entrepreneurship

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Pediatrics, Transplant and Cellular Therapy

pubs.publication-status

Published

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19

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