Cytosine arabinoside and mitoxantrone followed by second allogeneic transplant for the treatment of children with refractory juvenile myelomonocytic leukemia

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Abstract

Hematopoietic stem cell transplantation (HSCT) remains the only curative option for most patients with juvenile myelomonocytic leukemia (JMML). However, persistent disease and relapse rates after transplant range from 26% to 58%. We report the successful use of second HSCT after preparation with mitoxantrone and cytosine arabinoside (Ara-C) for patients with refractory or recurrent disease. Between 1993 and 2006, 5 children who underwent HSCT at our institution as initial therapy for JMML had persistent disease or relapsed. Pre-HSCT conditioning varied and donors were either HLA-matched siblings (n=2) or matched unrelated donors (n=3). After initial HSCT, they subsequently received high-dose Ara-C (3 g/m IV) every 12 hours on days -8 through -3 and mitoxantrone (10 mg/m/d IV) on days -8, -7, -6 followed by second HSCT from their original donors. All 5 patients are alive at 88, 179, 199, 234, and 246 months with no evidence of JMML, no significant toxicity, and 100% donor chimera as determined by PCR short-tandem repeat analysis. Our experience supports second transplant utilizing high-dose Ara-C and mitoxantrone in children with JMML who do not respond or relapse after first transplant.

Original languageEnglish (US)
Pages (from-to)491-494
Number of pages4
JournalJournal of Pediatric Hematology/Oncology
Volume36
Issue number6
DOIs
StatePublished - Jan 1 2014

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Juvenile Myelomonocytic Leukemia
Mitoxantrone
Hematopoietic Stem Cell Transplantation
Cytarabine
Transplants
Tissue Donors
Transplantation Conditioning
Therapeutics
Recurrence
Unrelated Donors
Microsatellite Repeats
Siblings
Polymerase Chain Reaction

Keywords

  • JMML
  • allogeneic transplant
  • chimerism

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

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title = "Cytosine arabinoside and mitoxantrone followed by second allogeneic transplant for the treatment of children with refractory juvenile myelomonocytic leukemia",
abstract = "Hematopoietic stem cell transplantation (HSCT) remains the only curative option for most patients with juvenile myelomonocytic leukemia (JMML). However, persistent disease and relapse rates after transplant range from 26{\%} to 58{\%}. We report the successful use of second HSCT after preparation with mitoxantrone and cytosine arabinoside (Ara-C) for patients with refractory or recurrent disease. Between 1993 and 2006, 5 children who underwent HSCT at our institution as initial therapy for JMML had persistent disease or relapsed. Pre-HSCT conditioning varied and donors were either HLA-matched siblings (n=2) or matched unrelated donors (n=3). After initial HSCT, they subsequently received high-dose Ara-C (3 g/m IV) every 12 hours on days -8 through -3 and mitoxantrone (10 mg/m/d IV) on days -8, -7, -6 followed by second HSCT from their original donors. All 5 patients are alive at 88, 179, 199, 234, and 246 months with no evidence of JMML, no significant toxicity, and 100{\%} donor chimera as determined by PCR short-tandem repeat analysis. Our experience supports second transplant utilizing high-dose Ara-C and mitoxantrone in children with JMML who do not respond or relapse after first transplant.",
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author = "Patel, {Sachit A} and Coulter, {Donald W} and Grovas, {Alfred C.} and Gordon, {Bruce Geoffrey} and Harper, {James Lloyd} and Warkentin, {Phyllis Irene} and Wisecarver, {James Lowell} and Sanger, {Warren G.} and Coccia, {Peter F.}",
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T1 - Cytosine arabinoside and mitoxantrone followed by second allogeneic transplant for the treatment of children with refractory juvenile myelomonocytic leukemia

AU - Patel, Sachit A

AU - Coulter, Donald W

AU - Grovas, Alfred C.

AU - Gordon, Bruce Geoffrey

AU - Harper, James Lloyd

AU - Warkentin, Phyllis Irene

AU - Wisecarver, James Lowell

AU - Sanger, Warren G.

AU - Coccia, Peter F.

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