@article {10766, title = {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.}, journal = {Cancers (Basel)}, volume = {10}, year = {2018}, month = {2018 Aug 27}, 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/m{\texttwosuperior}), 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.

}, issn = {2072-6694}, doi = {10.3390/cancers10090291}, author = {Grasso, Antonio G and Granzotto, Marilena and Zanon, Davide and Maestro, Alessandra and Loiacono, Stefano and Maximova, Natalia} } @article {10537, title = {Comparison of Efficacy and Safety of Caspofungin Versus Micafungin in Pediatric Allogeneic Stem Cell Transplant Recipients: A Retrospective Analysis.}, journal = {Adv Ther}, volume = {34}, year = {2017}, month = {2017 05}, pages = {1184-1199}, abstract = {

INTRODUCTION: The high morbidity and mortality associated with invasive fungal infections (IFIs) provide the rationale for antifungal prophylaxis in immuno-compromised pediatric patients undergoing hematopoietic stem cell transplantation (HSCT). Caspofungin and micafungin are antifungal agents of interest for prophylaxis of IFIs because of their potency against Candida and minimal toxicity or interactions with other drugs. Few studies have demonstrated the safety and efficacy of such echinocandins as prophylaxis for IFIs in patients undergoing HSCT.

METHODS: This retrospective cohort study compared caspofungin and micafungin for prevention of IFIs in 93 pediatric patients undergoing HSCT for oncological or non-oncological disease. The observation began with the first dose of antifungal agent and ended 3~months after transplantation.

RESULTS: Patients in the micafungin group had a higher overall treatment success rate of 87.2 versus 84.8\% in the caspofungin group, but the difference was not significant. There were no statistically significant differences in the incidence or type of proven/probable IFIs between the 2 groups. The low incidence of death did not differ statistically between the groups. Patients in the caspofungin group presented more frequently with fever, during and after neutropenia. In both groups, we observed an expected worsening of blood chemistry parameters. There were no adverse events definitely attributable to the two antifungal agents.

CONCLUSION: These results demonstrate good efficacy and tolerability for caspofungin and micafungin. However, better results with respect to the incidence and resolution of fever in the micafungin group may suggest its use in preference to that of caspofungin.

}, keywords = {Adolescent, Antifungal Agents, Caspofungin, Child, Child, Preschool, Cohort Studies, Echinocandins, Female, Hematopoietic Stem Cell Transplantation, Humans, Infant, Infant, Newborn, Lipopeptides, Male, Micafungin, Mycoses, Neutropenia, Retrospective Studies}, issn = {1865-8652}, doi = {10.1007/s12325-017-0534-7}, author = {Maximova, Natalia and Schillani, Giulia and Simeone, Roberto and Maestro, Alessandra and Zanon, Davide} } @article {3598, title = {Complete remission of VZV reactivation treated with valganciclovir in a patient with total lymphocyte depletion and acute kidney injury after allogeneic bone marrow transplantation.}, journal = {APMIS}, volume = {123}, year = {2015}, month = {2015 Jan}, pages = {77-80}, abstract = {

Varicella zoster virus (VZV), a threat for hematopoietic stem cell transplantation (HSCT) recipients, is still one of the most common viral pathogens that affect these patients with a reported incidence ranging between 17\% and 50\% in the post transplantation period. Valganciclovir (V-GCV), a valine ester pro-drug of GCV orally administrable, has recently shown great activity against CMV infections, but there are no reports of its clinical efficacy against VZV. We here report a case history of a patient with positive serologic test for VZV, who underwent allogeneic HSCT and developed an atypical varicella-like illness. First-line therapy with foscarnet had to be discontinued due rapid development of renal impairment (creatinine: 2.60 mg/dL, urea: 130.6 mg/dL) and therefore was switched to V-GCV. The renal impairment and skin lesions of the patient fully recovered after few days of therapy, even though the patient had complete lymphocyte depletion. This is the first case of a patient with chickenpox-like illness treated successfully with V-GCV.

}, keywords = {Acute Kidney Injury, Adolescent, Bone Marrow Transplantation, Fatal Outcome, Female, Ganciclovir, Herpes Zoster, Herpesvirus 3, Human, Humans, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Viral Load}, issn = {1600-0463}, doi = {10.1111/apm.12303}, author = {Maximova, Natalia and Antonio, Pizzol and Marilena, Granzotto and Rovere, Francesca and Tamaro, Paolo} }