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Open AccessCase Report

Complete Remission of a Refractory Acute Myeloid Leukemia with Myelodysplastic- and Monosomy 7-Related Changes after a Combined Conditioning Regimen of Plerixafor, Cytarabine and Melphalan in a 4-Year-Old Boy: A Case Report and Review of Literature

1
Department of Medicine, Surgery and Health Sciences, University of Trieste, Piazzale Europa 1, 34127 Trieste, Italy
2
Department of Laboratory Medicine, ASUITS, Piazza dell’Ospitale 1, 34129 Trieste, Italy
3
Pharmacy and Clinical Pharmacology Unit, Institute for Maternal and Child Health-IRCCS Burlo Garofolo, via dell’Istria 65/1, 34137 Trieste, Italy
4
Bone Marrow Transplant Unit, Institute for Maternal and Child Health-IRCCS Burlo Garofolo, via dell’Istria 65/1, 34137 Trieste, Italy
*
Author to whom correspondence should be addressed.
Cancers 2018, 10(9), 291; https://doi.org/10.3390/cancers10090291
Received: 19 July 2018 / Revised: 13 August 2018 / Accepted: 23 August 2018 / Published: 27 August 2018
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Abstract

Acute myeloid leukemia with myelodysplastic changes and monosomy 7 is a rare form of pediatric leukemia associated with very poor disease-free survival. The refractoriness of the disease is due to the protection offered by the bone marrow niche, making leukemic stem cells impervious to whatever chemotherapy or myeloablative regimen is chosen. Using a mobilizing agent for haematopoietic stem cells, Plerixafor, could sensitise leukemic cells to the myeloablative therapy. This approach was not previously used in a pediatric population, and in adult populations, was used in combination with busulphan with no difference in overall survival. We describe the case of a 4-year-old boy affected by refractory acute myeloid leukemia with myelodysplastic changes and monosomy 7. The child had never achieved a remission. We proposed a combined time-scheduled scheme of therapy with plerixafor and melphalan. Combining pharmacokinetics of plerixafor with pharmacokinetics and rapid and elevated myeloablative potential of melphalan in high dosage (200 mg/m2), we succeeded in mobilizing more than 85% of stem blasts immediately before infusion of Melphalan. The count of residual blasts after 8 h from melphalan infusion was only 1.3 cells/μL. The child achieved an engraftment at day +32 with full donor chimerism. Sixteen months after haematopoietic stem cell transplantation (HSCT), he is well and in complete remission. Our case suggests that the use of plerixafor before a conditioning therapy with melphalan could induce remission in acute myeloid leukemia refractory to the usual conditioning therapy in pediatric patients. This work adds strength to the body of knowledge regarding the “personalized” conditioning regimen for high-risk leukemic patients. View Full-Text
Keywords: acute myeloid leukemia; refractory; monosomy 7; pediatric; myelodysplastic; plerixafor; melphalan acute myeloid leukemia; refractory; monosomy 7; pediatric; myelodysplastic; plerixafor; melphalan
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Grasso, A.G.; Granzotto, M.; Zanon, D.; Maestro, A.; Loiacono, S.; Maximova, N. Complete Remission of a Refractory Acute Myeloid Leukemia with Myelodysplastic- and Monosomy 7-Related Changes after a Combined Conditioning Regimen of Plerixafor, Cytarabine and Melphalan in a 4-Year-Old Boy: A Case Report and Review of Literature. Cancers 2018, 10, 291.

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