Diffuse intrinsic pontine glioma (DIPG) remains a devastating disease. Panobinostat has been shown to have therapeutic efficacy both in vitro and in DIPG orthotopic xenograft models; however, clinical data in patients with DIPG are lacking. We present 2 cases of DIPG, who were treated with panobinostat at 22 to 25 mg/m2/dose, 3 times weekly for 2 weeks in 3-week cycles and concomitant reirradiation after disease progression. Two episodes of asymptomatic thrombocytopenia were observed in 1 patient. Hyperacetylation of histone H4 of peripheral blood mononuclear cells was evident following treatment. In our experience, panobinostat administered with reirradiation was well tolerated at a relatively higher dose than that used in adult studies.
Departments of *Pediatrics, Division of Pediatric Hematology Oncology
†Oncology, Molecular Therapeutics Program
∥Neurosurgery, Children’s Hospital of Michigan
#Radiation Oncology, Karmanos Cancer Institute, Wayne State University School of Medicine, Detroit, MI
¶Dana-Farber Boston Children’s Cancer and Blood Disorders Center, Boston, MA
The authors declare no conflict of interest.
Reprints: Zhihong J. Wang, MD, PhD, Department of Pediatrics, Division of Pediatric Hematology Oncology, Children’s Hospital of Michigan, 3901 Beaubien Street, Detroit, MI 48201 (e-mail: firstname.lastname@example.org).
Received July 24, 2016
Accepted January 26, 2017