Background
Aneurysmal bone cyst (ABC) is a benign bone tumor. Curettage and bone grafting is the common treatment. Here, we retrospectively evaluate nine patients treated with denosumab.
Procedure
...Nine patients with ABC, mostly pelvic and vertebral, treated with denosumab were analyzed retrospectively. A 70 mg/m2 denosumab dose was used weekly in the first month, and then monthly. Clinical and radiological responses to treatment were evaluated.
Results
In all patients, clinical symptoms including pain and limping regressed completely within 3 months. Radiological evaluation revealed changes in lesion size and content. In six patients, overall volume reduction in the range of 18–82% was detected. Decreases in the size and number of cysts were detected in eight patients. In five patients, fat signal appeared on follow‐up imaging. No major side effects were observed during treatment. Median follow‐up time after treatment was 15 months. At 5 months, severe hypercalcemia was observed in two patients due to rebound increase in osteoclastic activity. Subsequent to denosumab treatment, three patients underwent surgery for clinical or radiological recurrence.
Conclusions
Our results showed that denosumab provided a meaningful clinical and radiological improvement in ABC. It may be a treatment option, especially in spinal and pelvic tumors with potentially high surgical morbidity. However, late rebound hypercalcemia may restrict its use. Studies with more cases are required for routine use of denosumab in ABC.
Background
Lipoblastoma is a rare benign mesenchymal tumor of infancy and early childhood. Symptoms vary depending on localization, and signs of compression of adjacent organs may be seen.
Methods
A ...total of 12 children with pathologically proven lipoblastoma from 2000 to 2014 were reviewed retrospectively for their clinical features, treatment and follow up.
Results
There were seven boys and five girls between 7.5 months and 7.5 years of age. The most common symptom was painless rapid‐growing mass. The tumors were located in the neck, the abdomen, the groin, pelvis, axilla, glutea, labium majus, thigh and trunk. The largest tumor was in a retroperitoneal location and was 13 × 10 cm in size. Complete resection was performed in 10 patients. All of these patients were followed without any evidence of recurrence. Subtotal excision was performed in two patients. At a median follow up of 38 months (range, 1.8 months–10 years), all patients were disease free.
Conclusions
Lipoblastoma should be considered in the differential diagnosis of rapidly growing mass in patients <3 years of age. Total excision is the treatment of choice with preservation of vital organs. The prognosis is excellent despite large tumor size and local invasion. Recurrence rate is high in incompletely resected tumors. Regular follow up is important for early detection of recurrence.
Relapsed or refractory central nervous system (CNS) tumors still have poor prognosis, and, therefore, new treatment options are required. We retrospectively researched treatment results of patients ...with CNS tumors treated with nimotuzumab from 2010 to 2015. The study included nine patients with the diffuse intrinsic pontine glioma; eight with medulloblastoma; three each with anaplastic ependymoma, glioblastoma multiforme, and central nervous system primitive neuroectodermal tumor (CNS PNET); two patients with gliomatosis cerebri; and one patient each with other tumor types, including atypical teratoid rhabdoid tumor, thalamic astrocytoma, low-grade glial tumor, high-grade glial tumor, and cribriform neuroepithelial tumor. An objective response was observed in 10 of 33 patients with four patients showing a complete response, three a partial response, and three patients had stable disease. The 2-year overall survival (OS) and progression-free survival (PFS) rates were 35 ±9% and 19 ±8%, respectively. Due to the objective response in medulloblastoma, CNS PNET, and anaplastic ependymoma (MED group), survival rates of this group were analyzed. The 2-year OS and PFS for the MED group were 71 ±12% and 30 ±13%, respectively. The treatment was well tolerated. The treatment responses for medulloblastoma, CNS PNET, and anaplastic ependymoma have been promising. Likewise, some patients with relapsed or progressive CNS tumors may benefit through nimotuzumab-containing regimen.
The outcome of bone and soft tissue sarcomas (BST) after relapse has very poor prognosis with survival rates less than 39%. Unfortunately there are not many treatment options, but promising responses ...have been reported with ifosfamide, etoposide and carboplatin (ICE).
Therefore, we planned a study for children with recurrent/refractory BST treated with ICE regimen to evaluate their demographic features, responses to treatment and outcome.
Patients with primary diagnosis of BST and treated with ICE regimen at the time of first or subsequent relapse, progression or unresponsive disease were selected for study. The files were retrospectively evaluated.
Thirty of sixty-six patients had metastatic disease at relapse. Patients received median 5 cycles of ICE and were followed-up median 16.3 months. Overall survival rates were 83% and 62% at 1st and 2nd year. The ORR to the regimen was 43%. Survival rates were significantly higher in good responders (3-year EFS and OS rates: 50%
8% and 78%
14%; p<0.0001 and p<0.0001). Survival rates for non-metastatic disease were 42% and 75% for EFS and OS and higher than patients with metastases at relapse (p<0.001 and p<0.0001). EFS and OS rates of patients with rhabdomyosarcoma were 42% and 69%. No grade 3 or 4 renal toxicity was documented.
ICE combination improves the outcome of relapsed or refractory sarcomas and it might serve as second line treatment. Patients with no metastases at relapse would benefit more than others with metastatic disease.
The purpose of the present study was to examine maternal pain management in children with cancer and the associated factors.
The present work is a descriptive and cross-sectional study. Data for the ...study were obtained from mothers of children in the age group of 0 to 18 years undergoing treatment for solid tumors in Pediatric Oncology Service and Outpatient Clinics (n = 112). We used a questionnaire on parental pain management practices at home, the knowledge about pain and analgesic drugs, Spielberger State-Trait Anxiety Inventory (STAI), and Pain Catastrophizing Scale (PCS) to collect the data.
Several mothers taking part in the study reported various misconceptions about the assessment of children's pain, analgesic drugs, and usage of limited non-pharmacological methods for managing pain in children with cancer. No significant relationships were found between mothers' pain management practices, knowledge of pain assessment and analgesic drugs, and mothers' and children's sociodemographic characteristics or mothers' pain catastrophizing and anxiety about their own pain.
The findings of the study revealed that the majority of mothers of children with cancer had misconceptions regarding knowledge of pain assessment and analgesic drugs; these misconceptions potentially lead to manage children's pain associated with cancer ineffectively. Findings indicate mothers' information and support needs for children's cancer pain management in the home settings.
A further understanding of barriers to parental pain management in children with cancer in the home setting will contribute immensely in developing appropriate management practices.
•The present study is the first study to examine parental pain management practices and associated factors for children’cancer pain while at home in Turkey.•This study explores the relationship between mothers and children sociodemographic characteristics, mothers’ pain catastrophizing and anxiety about their own pain and parental pain management practices.•The majority of mothers apply to medical care when child had pain and very few mothers use nonpharmacological methods.•Many mothers have misconceptions of pain assessment and analgesic drugs.
Purpose
To evaluate the therapeutic outcome of intravitreal melphalan injection in the management of vitreous disease in patients with retinoblastoma. We particularly aimed to assess whether higher ...melphalan dose with lower number of injections was more effective and associated with fewer side effects.
Methods
This retrospective, interventional, noncomparative, and nonrandomized study included 39 eyes of 37 patients. Vitreous seeds were classified as dust, sphere, and cloud types. Intravitreal injections were performed through pars plana free of any visible tumor using 30-G needle. Response of the seeds (disappearance, conversion into inactive debris, or progression) and enucleation rate were determined as outcome measures.
Results
All patients previously received systemic or intra-arterial chemotherapy. Vitreous seeding was primary in 54% of eyes and secondary in 46% of eyes. Vitreous seeds were classified as dust in 9 (23.1%) eyes, sphere in 24 (61.5%) eyes, and cloud in 6 (15.4%) eyes. Melphalan dose varied between 20 and 40 µg and 20 (51.3%) eyes received >30 µg. The total number of injections was 70 (range 1-5, mean 1.8 per eye). Various types of regression were obtained in 27 (69.2%) eyes. Sphere-type seeds were the most responsive to melphalan. Nonresponse and disease progression were noted in 12 (30.8%) eyes. After a mean follow-up of 11.8 months, 17 (44%) eyes were enucleated. Vitreous hemorrhage (18%) and retinal pigment epithelial alterations (8%) were the most common side effects.
Conclusions
Intravitreal melphalan at 30-40 µg in 1 or 2 injections proved effective in 69.2% of eyes with vitreous disease.
Primary paraspinal/spinal epidural lymphoma (PPSEL) is rare in childhood. Here, we retrospectively evaluated patients with PPSEL treated in our department. We also reviewed the cases reported in the ...literature. Fifteen of 1354 non-Hodgkin lymphoma cases diagnosed over a 38-year period were PPSEPL. There were 11 male individuals and 4 female individuals with a median age of 13 years. Most common symptoms were pain and limb weakness. Physical examination revealed spinal cord compression in 80% of patients. The most common tumor location was the lumbar region. Histopathologic subtypes were lymphoblastic lymphoma in 6 and Burkitt lymphom in 5 patients. Subtotal or near-total excision of the tumor with laminectomy was performed in 6 patients. Thirteen and 9 patients received chemotherapy and radiotherapy, respectively. Neurologic recovery was observed in 70% of patients. Seven patients were alive without disease at a median of 88 months. Overall and event-free survival rates were 61.7% and 50.1%, respectively. We reviewed clinical features, treatment, and outcome of 69 PPSEL cases reported in the literature. Neurologic recovery and long-term survival was achieved in 66.7% of them. Heterogeneity in diagnostic methods and treatment have made it difficult to establish the prognostic indicators for neurologic outcome and survival. Multicenter prospective studies with more cases are necessary to determine the prognostic factors.
Purpose
To evaluate clinical outcomes and enucleation rates after intravitreal melphalan (IVM) alone and after IVM combined with intravitreal topotecan (IVT) for the treatment of vitreous disease, ...and to a lesser extent subretinal and retrohyaloid seeds, in patients with retinoblastoma.
Study design
A retrospective analysis of 77 eyes of 72 consecutive patients.
Methods
Demographic data, classification of tumors, seed type (dust, sphere or cloud) before injection and at the end of follow-up, injection type (IVM or IVM+IVT), doses of IVM and IVT, number of injections, follow-up time, enucleation status and side effects were recorded. Cox regression analysis and log-rank test for Kaplan-Meier curves were performed.
Results
Of 77 eyes, 40 received IVM alone (group 1) and 37 received IVM+IVT (group 2). Enucleation rates were 62.5% (n=25) in group 1 and 10.8% (n=4) in group 2 (p=0.001). Median eye survival was 23.6 months in group 1 and 25.6 months in group 2. Mantel-Cox test revealed statistically significant differences between Kaplan-Meier curves of group 1 and 2 (p=0.022). Multiple Cox regression analysis showed a significantly elevated enucleation rate associated with: IVM only treatment group (p=0.019) and pre-injection cloud type of seeding (p=0.014).
Conclusion
The combined use of intravitreal melphalan and topotecan provides significantly better results in terms of avoiding enucleation and vitreal and subretinal seed control.
