Desmoid-Type Fibromatosis in Children: A Step Forward in the Cooperative Group Setting

2012 ◽  
pp. 593-597
Author(s):  
Natalie Pounds ◽  
Stephen X. Skapek
Keyword(s):  
Clinical Trials ◽  
Desmoid Tumor ◽  
Aggressive Fibromatosis ◽  
Local Tumor Control ◽  
Therapeutic Interventions ◽  
Tumor Control ◽  
Group Setting ◽  
Soft Tissue Neoplasm ◽  
Life Threatening ◽  
Prospective Phase

Overview: Desmoid-type (aggressive) fibromatosis (desmoid tumor) is a soft tissue neoplasm that can occur in both children and adults. Although it is formally classified as an intermediate-grade neoplasm because of its propensity for locally invasive growth, it can lead to severe and life-threatening problems. Because metastases do not arise from desmoid tumor, therapeutic interventions have historically focused on surgery or radiation to achieve local tumor control. These approaches may be ineffective or impractical for some children. In those cases, systemic therapy with cytotoxic or noncytotoxic therapy has been used. Because of the relative rarity of this neoplasm in children, knowledge on the use of chemotherapy is based largely on anecdotal reports or retrospective series. Limited conclusions can be drawn, though, from these types of reports. In the last 10 years, two prospective phase II clinical trials of chemotherapy for children with desmoid tumor have been conducted in cooperative clinical trials centered in North America. We review the results of those clinical trials and suggest future directions for systematically approaching this disease to better define the role of chemotherapy for children with desmoid tumor.

scholarly journals A Prospective Phase II Study Evaluating Intraoperative Electrochemotherapy of Hepatocellular Carcinoma

Cancers ◽  
2020 ◽  
Vol 12 (12) ◽  
pp. 3778
Author(s):  
Mihajlo Djokic ◽  
Maja Cemazar ◽  
Masa Bosnjak ◽  
Rok Dezman ◽  
David Badovinac ◽  
...  
Keyword(s):  
Clinical Study ◽  
Phase Ii ◽  
Treatment Options ◽  
Progression Free Survival ◽  
Complete Response ◽  
Local Tumor Control ◽  
Tumor Control ◽  
Durable Response ◽  
Phase Ii Clinical Study ◽  
Prospective Phase

The aim of this clinical study was to investigate the effectiveness and long-term safety of electrochemotherapy as an emerging treatment for HCC in patients not suitable for other treatment options. A prospective phase II clinical study was conducted in patients with primary HCC who were not suitable for other treatment options according to the Barcelona Clinic Liver Cancer classification. A total of 24 patients with 32 tumors were treated by electrochemotherapy. The procedure was effective, feasible, and safe with some procedure-related side effects. The responses of the 32 treated nodules were: 84.4% complete response (CR), 12.5% partial response (PR), and 3.1% stable disease (SD). The treatment was equally effective for nodules located centrally and peripherally. Electrochemotherapy provided a durable response with local tumor control over 50 months of observation in 78.0% of nodules. The patient responses were: 79.2% CR and 16.6% PR. The median progression-free survival was 12 months (range 2.7–50), and the overall survival over 5 years of observation was 72.0%. This prospective phase II clinical study showed that electrochemotherapy was an effective, feasible, and safe option for treating HCC in patients not suitable for other treatment options.

scholarly journals Targeted therapy in advanced desmoid tumors: Current perspectives

2020 ◽  
Vol 11 (3) ◽  
pp. 9-16
Author(s):  
Amrallah A. Mohammed ◽  
Hani EL-Tanni ◽  
Hani M. EL-Khatib
Keyword(s):  
Targeted Therapy ◽  
Treatment Options ◽  
Aggressive Fibromatosis ◽  
Local Tumor Control ◽  
The Body ◽  
Treatment Modalities ◽  
Tumor Control ◽  
Desmoid Tumors ◽  
Treatment Needs ◽  
Medline Search

Abstract Background Desmoid tumors/aggressive fibromatosis (DTs/AF) are cytological bland fibrous neoplasms originating from the musculoaponeurotic structures throughout the body. The exact cause still remains unknown, however, they may present sporadically or as a manifestation of a hereditary syndrome called familial adenomatous polyposis (FAP). Although they lack the capacity to establish metastases, DTs/AF may be devastated and occasionally fatal. As a result of the heterogeneity of DTs/AF, treatment needs to be individualized to improve local tumor control and maintain patients’ quality of life. Therefore, after a multidisciplinary approach, all treatment options should be discussed with patients. Where systemic chemotherapy has been shown to be unsuccessful with marked side effects in case of advanced DTs/AF, new therapeutic options are needed. Methods A Medline search was conducted and published articles in different studies from 2000 to the present were reviewed. Conclusion More research is needed to illustrate both the prognostic and predictive factors of the targeted therapy and the value of their combinations with or without other treatment modalities to get the best result for the treatment of advanced DTs/AF.

Acute hepatic porphyrias for the neurologist: current concepts and perspectives

2021 ◽  
Author(s):  
Paulo Victor Sgobbi de Souza ◽  
Bruno de Mattos Lombardi Badia ◽  
Igor Braga Farias ◽  
Eduardo Augusto Gonçalves ◽  
Wladimir Bocca Vieira de Rezende Pinto ◽  
...  
Keyword(s):  
Central Nervous System Involvement ◽  
Therapeutic Interventions ◽  
Inborn Errors ◽  
Intravenous Glucose ◽  
Inherited Metabolic Disorders ◽  
Life Threatening ◽  
Acute Porphyrias ◽  
Diagnostic Work ◽  
Atypical Presentations ◽  
Interfering Rna

ABSTRACT Background: Acute hepatic porphyrias represent an expanding group of complex inherited metabolic disorders due to inborn errors of metabolism involving heme biosynthesis. Objective: We aimed to review the main clinical and therapeutic aspects associated with acute hepatic porphyrias. Methods: The authors provided a wide non-systematic review of current concepts and recently acquired knowledge about acute hepatic porphyrias. Results: Acute neurovisceral attacks are the most common and life-threatening presentation of this group and are often considered the main clinical manifestation by clinicians during differential diagnosis and the start of proper diagnostic work-up for acute porphyrias. However, atypical presentations with central nervous system involvement, neuropsychiatric disturbances, and some subtypes with photosensitivity usually make the definite diagnosis difficult and late. Early therapeutic interventions are essential during emergency treatment and intercritical periods to avoid recurrent severe presentations. The availability of new disease-modifying therapeutic proposals based on small interfering RNA (siRNA)-based therapies, complementary to the classic intravenous glucose infusion and hemin-based treatments, emphasizes the importance of early diagnosis and genetic counseling of patients. Conclusions: This review article highlights the main biochemical, pathophysiological, clinical, and therapeutic aspects of acute hepatic porphyrias in clinical practice.

Gamma Knife surgery combined with resection for treatment of a single brain metastasis: preliminary results

2010 ◽  
Vol 113 (Special_Supplement) ◽  
pp. 90-96 ◽  
Author(s):  
M. Yashar S. Kalani ◽  
Aristotelis S. Filippidis ◽  
Maziyar A. Kalani ◽  
Nader Sanai ◽  
David Brachman ◽  
...  
Keyword(s):  
Survival Data ◽  
Initial Treatment ◽  
Median Survival Time ◽  
Local Tumor Control ◽  
Survival Duration ◽  
Tumor Control ◽  
Local Tumor ◽  
Systemic Control ◽  
Cranial Metastasis

Object Resection and whole-brain radiation therapy (WBRT) have classically been the standard treatment for a single metastasis to the brain. The objective of this study was to evaluate the use of Gamma Knife surgery (GKS) as an alternative to WBRT in patients who had undergone resection and to evaluate patient survival and local tumor control. Methods The authors retrospectively reviewed the charts of 150 patients treated with a combination of stereotactic radiosurgery and resection of a cranial metastasis at their institution between April 1997 and September 2009. Patients who had multiple lesions or underwent both WBRT and GKS were excluded, as were patients for whom survival data beyond the initial treatment were not available. Clinical and imaging follow-up was assessed using notes from clinic visits and MR imaging studies when available. Follow-up data beyond the initial treatment and survival data were available for 68 patients. Results The study included 37 women (54.4%) and 31 men (45.6%) (mean age 60 years, range 28–89 years). In 45 patients (66.2%) there was systemic control of the primary tumor when the cranial metastasis was identified. The median duration between resection and radiosurgery was 15.5 days. The median volume of the treated cavity was 10.35 cm3 (range 0.9–45.4 cm3), and the median dose to the cavity margin was 15 Gy (range 14–30 Gy), delivered to the 50% isodose line (range 50%–76% isodose line). The patients' median preradiosurgery Karnofsky Performance Scale (KPS) score was 90 (range 40–100). During the follow-up period we identified 27 patients (39.7%) with recurrent tumor located either local or distant to the site of treatment. The median time from primary treatment of metastasis to recurrence was 10.6 months. The patients' median length of survival (interval between first treatment of cerebral metastasis and last follow-up) was 13.2 months. For the patient who died during follow-up, the median time from diagnosis of cerebral metastasis to death was 11.5 months. The median duration of survival from diagnosis of the primary cancer to last follow-up was 30.2 months. Patients with a pretreatment KPS score ≥ 90 had a median survival time of 23.2 months, and patients with a pretreatment KPS score < 90 had a median survival time of 10 months (p < 0.008). Systemic control of disease at the time of metastasis was not predictive of increased survival duration, although it did tend to improve survival. Conclusions Although the debate about the ideal form of radiation treatment after resection continues, these findings indicate that GKS combined with surgery offers comparable survival duration and local tumor control to WBRT for patients with a diagnosis of a single metastasis.

Petroclival meningiomas: long-term outcomes of multimodal treatments and management strategies based on 30 years of experience at a single institution

2020 ◽  
Vol 132 (6) ◽  
pp. 1675-1682 ◽  
Author(s):  
Jin Wook Kim ◽  
Hee-Won Jung ◽  
Yong Hwy Kim ◽  
Chul-Kee Park ◽  
Hyun-Tai Chung ◽  
...  
Keyword(s):  
Adjuvant Treatment ◽  
Management Strategies ◽  
Local Tumor Control ◽  
Tumor Control ◽  
Years Of Experience ◽  
Local Tumor ◽  
Long Term Outcomes ◽  
Single Institution ◽  
Petroclival Meningiomas

OBJECTIVEA thorough investigation of the long-term outcomes and chronological changes of multimodal treatments for petroclival meningiomas is required to establish optimal management strategies. The authors retrospectively reviewed the long-term clinical outcomes of patients with petroclival meningioma according to various treatments, including various surgical approaches, and they suggest treatment strategies based on 30 years of experience at a single institution.METHODSNinety-two patients with petroclival meningiomas were treated surgically at the authors’ institution from 1986 to 2015. Patient demographics, overall survival, local tumor control rates, and functional outcomes according to multimodal treatments, as well as chronological change in management strategies, were evaluated. The mean clinical and radiological follow-up periods were 121 months (range 1–368 months) and 105 months (range 1–348 months), respectively.RESULTSA posterior transpetrosal approach was most frequently selected and was followed in 44 patients (48%); a simple retrosigmoid approach, undertaken in 30 patients, was the second most common. The initial extent of resection and following adjuvant treatment modality were classified into 3 subgroups: gross-total resection (GTR) only in 13 patients; non-GTR treatment followed by adjuvant radiosurgery or radiation therapy (non-GTR+RS/RT) in 56 patients; and non-GTR without adjuvant treatment (non-GTR only) in 23 patients. The overall progression-free survival rate was 85.8% at 5 years and 81.2% at 10 years. Progression or recurrence rates according to each subgroup were 7.7%, 12.5%, and 30.4%, respectively.CONCLUSIONSThe authors’ preferred multimodal treatment strategy, that of planned incomplete resection and subsequent adjuvant radiosurgery, is a feasible option for the management of patients with large petroclival meningiomas, considering both local tumor control and postoperative quality of life.

Combination of microsurgery and Gamma Knife surgery for the treatment of intracranial chondrosarcomas

2006 ◽  
Vol 105 (Supplement) ◽  
pp. 18-25 ◽  
Author(s):  
Petter Förander ◽  
Tiit Rähn ◽  
Lars Kihlström ◽  
Elfar Ulfarsson ◽  
Tiit Mathiesen
Keyword(s):  
Gamma Knife ◽  
Cranial Nerve ◽  
Residual Tumor ◽  
Local Tumor Control ◽  
Gamma Knife Surgery ◽  
Long Term Results ◽  
Tumor Control ◽  
Low Grade ◽  
Cranial Nerve Palsies ◽  
Tumor Volume Reduction

ObjectIntracranial chondrosarcomas have a high risk of recurrence after surgery. This retrospective study of patients with intracranial chondrosarcoma was conducted to determine the long-term results of microsurgery followed by Gamma Knife surgery (GKS) for residual tumor or recurrence.MethodsThe authors treated nine patients whose median age was 36 years. Seven patients had low-grade chondrosarcomas (LGCSs), whereas mesenchymal chondrosarcomas (MCSs) were diagnosed in two. Radiosurgery was performed in eight patients, whereas one patient declined further surgical intervention and tumor-volume reduction necessary for the GKS.The patients were followed up for 15 to 173 months (median 108 months) after diagnosis and 3 to 166 months (median 88 months) after GKS. Seven patients had residual tumor tissue after microsurgery, and two operations appeared radical. In the two latter cases, tumors recurred after 25 and 45 months. Thus, definite tumor control was not achieved after surgery alone in any patient, whereas the addition of radiosurgery allowed tumor control in all six patients with LGCSs. Two of these patients experienced an initial tumor regrowth after GKS; in both cases the recurrences were outside the prescribed radiation field. The patients underwent repeated GKS, and subsequent tumor control was observed. An MCS was diagnosed in the remaining two patients. Complications after microsurgery included diplopia, facial numbness, and paresis. After GKS, one patient had radiation necrosis, which required microsurgery, and two patients had new cranial nerve palsies.Conclusions Tumor control after microsurgery alone was not achieved in any patient, whereas adjuvant radiosurgery provided local tumor control in six of eight GKS-treated patients. Tumor control was not achieved in the two patients with MCS. Similar to other treatments for intracranial chondrosarcoma, morbidity after micro- and radiosurgical combination therapy was high and included severe cranial nerve palsies.

The Epitome of Novel Techniques and Targeting Approaches in Drug Delivery for Treating Lymphatic Filariasis

2020 ◽  
Vol 21 (12) ◽  
pp. 1250-1263
Author(s):  
Saurabh Shrivastava ◽  
Anshita Gupta ◽  
Chanchal Deep Kaur
Keyword(s):  
Lymphatic Filariasis ◽  
Neglected Tropical Diseases ◽  
Therapeutic Interventions ◽  
Tropical Diseases ◽  
Human Beings ◽  
Therapeutic Approaches ◽  
Preventive Chemotherapy ◽  
Bioactive Substances ◽  
Lymphatic Diseases ◽  
Life Threatening

Background: Lymphatic filariasis is a pervasive and life-threatening disease for human beings. Currently, 893 million people in 49 countries worldwide affected by lymphatic filariasis as per WHO statistics. The concealed aspects of lymphatic diseases such as delayed disease detection, inappropriate disease imaging, the geographical outbreak of infection, and lack of preventive chemotherapy have brought this epidemic to the edge of Neglected Tropical Diseases. Many medications and natural bioactive substances have seen to promote filaricidal activity against the target parasitic species. However, the majority of failures have occurred in pharmaceutical and pharmacokinetic issues. Objective: The purpose of the study is to focus on the challenges and therapeutic issues in the treatment of filariasis. The review brings novel techniques and therapeutic approaches for combating lymphatic filariasis. It also offers significant developments and opportunities for such therapeutic interventions. Conclusion: Through this review, an attempt has made to critically evaluate the avenues of innovative pharmaceuticals and molecular targeting approaches to bring an integrated solution to combat lymphatic filariasis.

scholarly journals Stereotactic radiosurgery for treating meningiomas eligible for complete resection

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Maximilian I. Ruge ◽  
Juman Tutunji ◽  
Daniel Rueß ◽  
Eren Celik ◽  
Christian Baues ◽  
...  
Keyword(s):  
Adverse Events ◽  
Stereotactic Radiosurgery ◽  
Medical Condition ◽  
Local Tumor Control ◽  
Complete Resection ◽  
Neurological Deficits ◽  
Tumor Control ◽  
Recurrence Rates ◽  
Treatment Volume

Abstract Background For meningiomas, complete resection is recommended as first-line treatment while stereotactic radiosurgery (SRS) is established for meningiomas of smaller size considered inoperable. If the patient´s medical condition or preference excludes surgery, SRS remains a treatment option. We evaluated the efficacy and safety of SRS in a cohort comprising these cases. Methods In this retrospective single-centre analysis we included patients receiving single fraction SRS either by modified LINAC or robotic guidance by Cyberknife for potentially resectable intracranial meningiomas. Treatment-related adverse events as well as local and regional control rates were determined from follow-up imaging and estimated by the Kaplan–Meier method. Results We analyzed 188 patients with 218 meningiomas. The median radiological, and clinical follow-up periods were 51.4 (6.2–289.6) and 55.8 (6.2–300.9) months. The median tumor volume was 4.2 ml (0.1–22), and the mean marginal radiation dose was 13.0 ± 3.1 Gy, with reference to the 80.0 ± 11.2% isodose level. Local recurrence was observed in one case (0.5%) after 239 months. The estimated 2-, 5-, 10- and 15-year regional recurrence rates were 1.5%, 3.0%, 6.6% and 6.6%, respectively. Early adverse events (≤ 6 months after SRS) occurred in 11.2% (CTCEA grade 1–2) and resolved during follow-up in 7.4% of patients, while late adverse events were documented in 14.4% (grade 1–2; one case grade 3). Adverse effects (early and late) were associated with the presence of symptoms or neurological deficits prior to SRS (p < 0.03) and correlated with the treatment volume (p < 0.02). Conclusion In this analysis SRS appears to be an effective treatment for patients with meningiomas eligible for complete resection and provides reliable long-term local tumor control with low rates of mild morbidity.

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