Danuta Perek

Effective everolimus treatment of inoperable, life-threatening subependymal giant cell astrocytoma and intractable epilepsy in a patient with tuberous sclerosis complex

We present successful everolimus treatment of a huge subependymal giant cell astrocytoma in a 10-year old boy with tuberous sclerosis complex. The patient underwent several partial tumor resections complicated by intraoperative cardiac arrest. The tumor has been regrowing and produced severe clinical symptoms. Everolimus treatment resulted in marked tumor regression, significant improvement in patients ambulation and cessation of seizures. Moreover, the therapy was well tolerated. These findings indicate that everolimus treatment should be considered as a therapeutic option alternative to surgery in patients with tuberous sclerosis complex.

Risk factors of recurrence in 157 MB/PNET patients treated in one institution

Abstract To evaluate the risk factors for recurrence of MB/PNET we analyzed the medical records of 157 patients treated at the Childrens Memorial Health Institute between February 1981 and February 1997. The following factors were evaluated: age at diagnosis, gender, tumor size, tumor cells in the CSF, postoperative status, extent of resection and methods of treatment. We evaluated chemotherapy (CHT) doses, interval between courses, interval between surgery (S) and first course of CHT, interval between S and radiotherapy (RTX), and breaks during RTX. We divided patients into six groups: S alone, S+CHT, S+RTX, S+CHT+ RTX, S+RTX+CHT, S+CHT+RTX+ CHT. Age at diagnosis, gender, tumor size, extent of resection, postoperative status, intervals between courses of CHT, between S and the first course of CHT, and between S and RTX, and breaks during RTX had no statistical influence on relapse occurrence. Tumor cells in CSF were routinely checked for from January 1992 onward. In this group of 75 patients, 40 had tumor cells positive at surgery (28 relapsed), while in the group of 35 patients with negative tumor cells 14 relapsed (P=0.004). Out of 26 patients treated with S+RTX alone, 13 relapsed. Among 14 patients treated with S+RTX and prolonged CHT 6 relapsed. Out of 14 patients treated with S+CHT 13 relapsed; among 49 who received S+CHT+RTX 35 relapsed; and out of 51 patients treated with S+CHT+RTX+CHT 30 relapsed. In the multivariate analysis of treatment methods chemotherapy implemented after radiotherapy had a positive, though not statistically significant, influence on outcome (P=0.06). Among those receiving CHT the mean percentage of the ideal dose administered had a statistically significant influence on relapse: in the group of relapsed patients the mean dose was 76.1%, while in the group in continuous remission it was 83.7% (P=0.0013). On the basis of our data, we conclude that the presence of tumor cells in the CSF had a significant influence on the occurrence of relapse. Administration of appropriate doses of chemotherapy is extremely important for the occurrence of relapse and the final outcome of treatment. Prolonged adjuvant chemotherapy after radiotherapy seems to lower the risk of recurrence.

Micafungin in invasive fungal infections in children with acute leukemia or undergoing stem cell transplantation

Jan Styczynski, Krzysztof Czyzewski, Mariusz Wysocki, Olga Zajac-Spychala, Jacek Wachowiak, Tomasz Ociepa, Tomasz Urasinski, Olga Gryniewicz-Kwiatkowska, Agnieszka Kolodziejczyk-Gietka, Bozenna Dembowska-Baginska, Danuta Perek, Malgorzata Salamonowicz, Lukasz Hutnik, Michal Matysiak, Karolina Siewiera, Jowita Frackiewicz, Krzysztof Kalwak, Wanda Badowska, Zofia Malas, Jolanta Gozdzik, Agnieszka Urbanek-Dadela, Graz_yna Karolczyk, Weronika Stolpa, Grazyna Sobol, Zuzanna Gamrot, Mariola Woszczyk and Lidia Gil Department of Pediatric Hematology and Oncology, Collegium Medicum, Nicolaus Copernicus University Torun, Bydgoszcz, Poland; Department of Pediatric Oncology, Hematology and Transplantology, University of Medical Sciences, Poznan, Poland; Department of Pediatric Hematology and Oncology, Pomeranian Medical University, Szczecin, Poland; Department of Oncology, Children’s Memorial Health Institute, Warszawa, Poland; Department of Pediatric Hematology and Oncology, Medical University, Warszawa, Poland; Department of Pediatric Stem Cell Transplantation, Hematology and Oncology, Medical University, Wroclaw, Poland; Division of Pediatric Hematology and Oncology, Children Hospital, Olsztyn, Poland; Department of Clinical Immunology and Transplantology, Stem Cell Transplant Center, University Children’s Hospital, Jagiellonian University Collegium Medicum, Krakow, Poland; Division of Pediatric Hematology and Oncology, Children Hospital, Kielce, Poland; Department of Pediatric, Division of Pediatric Oncology, Hematology and Chemotherapy, Silesian Medical University, Katowice, Poland; Division of Paediatric Haematology and Oncology, Chorzow Paediatric and Oncology Center, Chorzow, Poland; Department of Hematology, University of Medical Sciences, Poznan, Poland

Trilateral Retinoblastoma: Diagnosis Using Magnetic Resonance Imaging

Retinoblastoma is the most common pediatric intraocular neoplasm and accounts for ∼3% of pediatric malignant tumors, affecting approximately 1 in 18,000 children under 5 years of age in the U.S. It is a highly malignant tumor of the primitive neural retina. Histologically, retinoblastoma develops from immature retinal cells and replaces the retina and other intraocular tissues. Neoplastic cell proliferation is caused by the inactivation of both copies of a tumor suppressor gene (Rb1) that participates in the control of cell cycling. Occasionally patients with ocular hereditary retinoblastoma have an associated independent primary midline intracranial neuroblastic tumor. This syndrome is called trilateral retinoblastoma. Midline intracranial tumors are typically located in the pineal region, but occurrences in the sellar and suprasellar regions are also found. The prognosis for trilateral retinoblastoma is markedly worse, with a high rate of subarachnoid tumor spread. The mean survival in this group of patients after treatment is about 9 months. An association between pineal cysts and hereditary retinoblastoma has been described in the recent literature, found that the prevalence of pineal cysts in children with bilateral, hereditary retinoblastoma was statistically significant compared with that in patients with unilateral retinoblastoma. Pineal cysts are supposed to be a benign variant of trilateral retinoblastoma. MR imaging has become a very useful diagnostic tool in evaluation of patients with retinoblastoma. MR is the imaging modality of choice in detection of leptomeningeal spread of the tumor and evaluation of primary intracranial tumors that can be associated with retinoblastoma. On the basis of some cases in which intracranial midline tumors were diagnosed earlier than intraocular lesions, we strongly recommend very careful ophthalmologic and imaging examinations of orbits in all children below 4 years of age with a intracranial midline primary tumor.