title: High-dose chemotherapy followed by autologous hematopoietic stem cell transplantation for adult histiocytic disorders with central nervous system involvement.
abstract: We postulated that high-dose chemotherapy (HDC) followed by peripheral autologous hematopoietic stem cell transplantation might help to control refractory central nervous system (CNS) histiocytic disorders. Six patients with histiocytic CNS involvement were treated in this way. Two patients achieved non-active disease status, although one relapsed at 84 months. Two patients had regressive disease, one of whom progressed at 21 months. One patient had progressive disease at 14 months. One patient had extra-CNS progression but CNS regression. After a median follow-up of 22.4 months, only one of the six patients still has non-active disease. Treatment was effective on craniofacial and space-occupying brainstem lesions, and was ineffective on neurodegenerative lesions.
authors.affiliation: Service d'Hématologie Clinique Hôpital La Pitié-Salpétrière 47-83 boulevard de l'Hôpital, 75013 Paris, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Gaspar
authors.forename: Pascaline
authors.initials: P
authors.lastname: Boudou
authors.forename: Julien
authors.initials: J
authors.lastname: Haroche
authors.forename: Bertrand
authors.initials: B
authors.lastname: Wechsler
authors.forename: Eric
authors.initials: E
authors.lastname: Van Den Neste
authors.forename: Khe
authors.initials: K
authors.lastname: Hoang-Xuan
authors.forename: Zahir
authors.initials: Z
authors.lastname: Amoura
authors.forename: Remy
authors.initials: R
authors.lastname: Guillevin
authors.forename: Julien
authors.initials: J
authors.lastname: Savatovski
authors.forename: Nabih
authors.initials: N
authors.lastname: Azar
authors.forename: Jean-Charles
authors.initials: JC
authors.lastname: Piette
authors.forename: Véronique
authors.initials: V
authors.lastname: Leblond
mesh_terms: D000328:Adult; D003131:Combined Modality Therapy; D004305:Dose-Response Relationship, Drug; D015614:Histiocytosis / Q000188:drug therapy* / Q000628:therapy*; D006801:Humans; D008875:Middle Aged; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome