title: High-dose chemotherapy with autologous stem cell rescue for treatment of retinoblastoma: report of five cases.
abstract: RB is a primarily pediatric cancer arising from the retina, initiated by biallelic loss of the RB1 gene. We report five children with bilateral RB (n = 3), extra-ocular disseminated RB, or disseminated relapsed RB, who were treated with tandem high-dose chemotherapy and autologous stem cell rescue. All patients received at least 2.2 × 10(6) /kg CD34(+) (median, 3.9 × 10(6) /kg) cells. The preparative regimen for course 1 was carboplatin, thiotepa, etoposide, and for course 2, CM and melphalan. ANC of at least 0.5 × 10(9) /L occurred at a median of 11 days (range, 10-12) and 15 days (range, 12-16) after the first and second procedure, respectively. Platelet engraftment occurred at a median of 13 days (range, 12-17) and 15 days (range, 14-22) after the first and second procedure, respectively. All of the five patients treated remain alive and disease free at the last follow-up time, ranging between 21 and 44 months after completion of autologous transplant. Additional therapy was required in one patient, in whom enucleation had to be performed because of early disease relapse, refractory to local therapy. Intensification of chemotherapy with repeated high-dose chemotherapy and autologous rescue appears an acceptable choice in selected cases with bilateral or extra-ocular disease, either recurrent or refractory.
authors.affiliation: Medical Direction, Azienda Ospedaliero Universitaria Meyer Children Hospital, Firenze, Italy.
authors.forename: Désirée
authors.initials: D
authors.lastname: Caselli
authors.forename: Angela
authors.initials: A
authors.lastname: Tamburini
authors.forename: Agostino
authors.initials: A
authors.lastname: La Torre
authors.forename: Liliana
authors.initials: L
authors.lastname: Pollazzi
authors.forename: Veronica
authors.initials: V
authors.lastname: Tintori
authors.forename: Franco
authors.initials: F
authors.lastname: Bambi
authors.forename: Roberto
authors.initials: R
authors.lastname: Caputo
authors.forename: Maurizio
authors.initials: M
authors.lastname: Aricò
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D012175:Retinoblastoma / Q000628:therapy*; D014182:Transplantation, Autologous
keywords: autologous stem cell transplant; enucleation; retinoblastoma