title: Successful autologous peripheral blood stem cell transplantation using thiotepa in a patient with systemic sclerosis and cardiac involvement.
abstract: A 19-year-old man with systemic sclerosis (SSc) was hospitalized for autologous peripheral blood stem cell transplantation (auto-PBSCT) due to progressive scleroderma and cardiac involvement despite conventional treatment. During the administration of cyclophosphamide (60 mg/kg/day for 2 days) for mobilization and collection of CD34+ selected peripheral blood stem cells, he developed congestive heart failure. Echocardiogram showed hypokinetic asynergy from the septum to posterior wall, which might indicate underlying cardiac damage. We were also concerned about the risk of high-dose cyclophosphamide-induced cardiotoxicity. Since the dose-limiting toxicity of thiotepa, an alkylating agent, is myelosuppression, and cardiac toxicity due to thiotepa is less common, we used a conditioning regimen consisting of thiotepa (10 mg/kg/day, day -5) and low-dose cyclophosphamide (50 mg/kg/day, days -3 and -2), instead of the conventional high-dose cyclophosphamide (50 mg/kg/day x 4 days/course). The post-transplant course was uneventful, and the modified Rodnan skin thickness score improved from 32 to 15. The present case report demonstrates that thiotepa can be employed as a conditioning regimen for auto-PBSCT in SSc patients with cardiac involvement in order to reduce cyclophosphamide-induced cardiotoxicity.
authors.affiliation: Third Department of Internal Medicine, Akita University School of Medicine, 1-1-1 Hondo, Akita City, Akita 010-8543, Japan. komatsud@med.akita-u.ac.jp
authors.forename: Atsushi
authors.initials: A
authors.lastname: Komatsuda
authors.forename: Yoshinari
authors.initials: Y
authors.lastname: Kawabata
authors.forename: Takahito
authors.initials: T
authors.lastname: Horiuchi
authors.forename: Mutsuhito
authors.initials: M
authors.lastname: Motegi
authors.forename: Masatoyo
authors.initials: M
authors.lastname: Ozawa
authors.forename: Naohito
authors.initials: N
authors.lastname: Fujishima
authors.forename: Masaaki
authors.initials: M
authors.lastname: Kume
authors.forename: Makoto
authors.initials: M
authors.lastname: Hirokawa
authors.forename: Hideki
authors.initials: H
authors.lastname: Wakui
authors.forename: Akihiko
authors.initials: A
authors.lastname: Yamaguchi
authors.forename: Ken-ichi
authors.initials: K
authors.lastname: Sawada
mesh_terms: D000328:Adult; D018906:Antineoplastic Agents, Alkylating / Q000494:pharmacology*; D003520:Cyclophosphamide / Q000009:adverse effects / Q000494:pharmacology; D006331:Heart Diseases / Q000139:chemically induced / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists / Q000494:pharmacology*; D012595:Scleroderma, Systemic / Q000503:physiopathology / Q000628:therapy*; D033581:Stem Cell Transplantation; D013852:Thiotepa / Q000494:pharmacology*; D013997:Time Factors; D014182:Transplantation, Autologous
chemical_list: D018906:Antineoplastic Agents, Alkylating; D019653:Myeloablative Agonists; D003520:Cyclophosphamide; D013852:Thiotepa