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

Kris M. Mahadeo; Priti Tewari; Suhag H. Parikh; Timothy A. Driscoll; Kristin Page; Paul L. Martin; Joanne Kurtzberg; Vinod K. Prasad

doi:10.1111/petr.12577

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

Kris M. Mahadeo, Priti Tewari, Suhag H. Parikh, Timothy A. Driscoll, Kristin Page, Paul L. Martin, Joanne Kurtzberg, Vinod K. Prasad

Research output: Contribution to journal › Article › peer-review

10 Scopus citations

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.

Original language	English (US)
Pages (from-to)	753-757
Number of pages	5
Journal	Pediatric Transplantation
Volume	19
Issue number	7
DOIs	https://doi.org/10.1111/petr.12577
State	Published - 2015
Externally published	Yes

Keywords

congenital amegakaryocytic thrombocytopenia
cord blood transplantation
hematopoietic stem cell transplantation
thrombocytopenia

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Transplantation

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10.1111/petr.12577

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Mahadeo, K. M., Tewari, P., Parikh, S. H., Driscoll, T. A., Page, K., Martin, P. L., Kurtzberg, J., & Prasad, V. K. (2015). Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation. Pediatric Transplantation, 19(7), 753-757. https://doi.org/10.1111/petr.12577

Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation. / Mahadeo, Kris M.; Tewari, Priti; Parikh, Suhag H. et al.
In: Pediatric Transplantation, Vol. 19, No. 7, 2015, p. 753-757.

Research output: Contribution to journal › Article › peer-review

Mahadeo, KM, Tewari, P, Parikh, SH, Driscoll, TA, Page, K, Martin, PL, Kurtzberg, J & Prasad, VK 2015, 'Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation', Pediatric Transplantation, vol. 19, no. 7, pp. 753-757. https://doi.org/10.1111/petr.12577

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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.",

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

Abstract

Keywords

ASJC Scopus subject areas

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