scholarly journals MON-098 Risk of Long-Term Endocrine Sequelae in Survivors of Progressing Childhood Optic Pathway Glioma Treated by Upfront Chemotherapy: Preliminary Analyses of 102 Subjects from the French Multicentric BB-SFOP Registry

2020 ◽  
Vol 4 (Supplement_1) ◽  
Author(s):  
Helene Hippolyte ◽  
Emilie De Carli ◽  
Isabelle Pellier ◽  
Xavier Rialland ◽  
Regis Coutant

Abstract For the brain tumor committee of SFCE (Societe ́ Française des Cancers de l’Enfant). Objective: Therapeutic approach favors chemotherapy as the first-line-treatment in progressing OPG. There are few data on long term endocrine outcomes of aggressive OPG treated by upfront chemotherapy. Our main objective was to describe the long-term endocrine sequelae in these patients and to identify potential early predictors of the endocrine involvement. Subjects and methods: Children diagnosed with OPG at an age younger than 16 years from the French multicentric BBSFOP registry were included. They were treated with upfront chemotherapy according to the BB-SFOP protocol in France between June 1990 and December 2004, and subsequent treatment (second-line chemotherapy, surgery, radiotherapy) was used depending on tumor progression. They underwent a late evaluation with clinical and biological assessment between January 2011 and March 2016. Results: One hundred and two patients were included in our study. The mean age at tumor diagnosis was 3.3±0.3 years. The mean time of follow-up was 13.9±3.7 years. A history of precocious puberty was present in 36% of the subjects. At least one endocrine deficiency was present in 93% of the subjects (GHD 74%, TSH deficiency 57%, ACTH deficiency 36%, hypogonadotropism 33%, gonadic deficiency 30%, diabetes insipidus 15%; inappropriate AVP secretion 7%). 37% of males and 39% of females were overweight or obese. Mean adult height, reached in 51 subjects, was -1.2±1.3 SDS in males, and -0.7±1.4 SDS in females. Chemotherapy only was protective from pituitary deficiencies (odds ratio 0.19 to 0.37, P < 0.05). NF1 was protective from TSH and ACTH deficiencies (odds ratio 0.25 to 0.35, P < 0.05). Tumor volume on diagnostic MRI was not predictive of pituitary deficiencies. Gonadic deficiency was significantly more frequent in males than females (46,5% vs 12.2%, P < 0.05), and associated with chemotherapy only (OR 3.2, P < 0.05) and NF1 (OR 4.8, P < 0.05). Overweight/Obesity was associated with ACTH deficiency (OR 5, P < 0.05).Conclusion: Obesity and late endocrine dysfunction were frequent in subjects treated by upfront chemotherapy for aggressive OPG during childhood. However, chemotherapy only, when possible, was protective from pituitary involvement.

2021 ◽  
pp. 088307382110531
Author(s):  
Cemal Karakas ◽  
Emin Fidan ◽  
Kapil Arya ◽  
Troy Webber ◽  
Joan B. Cracco

To determine the frequency, predictors, and outcomes of seizures in patients with myelomeningocele, we retrospectively analyzed the data from patients with myelomeningocele followed longitudinally at a single center from 1975 to 2013. We identified a total of 122 patients (61% female). The mean follow-up duration was 11.1 years (minimum-maximum = 0-34.5 years, SD = 8.8, median = 9.1 years). A total of 108 (88.5%) patients had hydrocephalus, and 98 (90.7%) of those patients required a ventriculoperitoneal shunt procedure. Twenty-four (19.7%) patients manifested with seizures, 23 of whom had hydrocephalus. The average age of seizure onset was 4.8 years (median 2 years of age). Falx dysgenesis ( P = .004), lumbar myelomeningocele ( P = .007), and cortical atrophy ( P = .028) were significantly associated with epileptic seizure development. The average seizure-free period at the last follow-up in patients with a history of myelomeningocele and seizures was 8.1 years. We conclude that myelomeningocele patients with seizures have an overall good prognosis with considerable long-term seizure freedom.


2016 ◽  
Vol 07 (S 01) ◽  
pp. S026-S030 ◽  
Author(s):  
Vaios Peritogiannis ◽  
Thiresia Manthopoulou ◽  
Venetsanos Mavreas

ABSTRACT Introduction: Long-term benzodiazepine (BZD) treatment in patients with mental disorders is widespread in clinical practice, and this is also the case of patients with schizophrenia, although the evidence is weak and BZD prescription is discouraged by guidelines and medical authorities. Data on BZD prescription are usually derived from national or regional databases whereas information on the use of BZD by patients with schizophrenia and related psychoses in general population-based samples is limited. Materials and Methods: Information for 77 patients with psychotic disorders who were regularly attending follow-up appointments with the multidisciplinary Mobile Mental Health Unit of the prefectures of Ioannina and Thesprotia, Northwest Greece, during 1-year period (2015) was obtained from our database. Results: From the total of 77 engaged patients, 30 (39%) were regularly prescribed BZDs in the long term, as part of their treatment regimen. Prescribed BZDs were mostly diazepam and lorazepam, in 43.3% of cases each. The mean daily dose of these compounds was 13 mg and 3.77 mg, respectively. Statistical analysis showed a correlation of long-term BZD use with the history of alcohol/substance abuse. Most patients were receiving BZD continuously for several years, and the mean dose was steady within this interval. Conclusions: A large proportion of patients with psychotic disorders were regularly prescribed BZD in long term. It appears that when BZDs are prescribed for some period in the course of a psychotic disorder, their use commonly exceeds the recommended interval and then becomes a regular part of the chronic treatment regimen. Future research should address the factors that may be related to the long-term BZD use by patients with psychotic disorders. Interventions for the reduction of regular BZD prescription should target the primary care setting and all those who treat first episode patients.


Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2338-2338
Author(s):  
Lena Coïc ◽  
Suzanne Verlhac ◽  
Emmanuelle Lesprit ◽  
Emmanuelle Fleurence ◽  
Francoise Bernaudin

Abstract Abnormal TCD defined as high mean maximum velocities > 200 cm/sec are highly predictive of stroke risk and justify long term transfusion program. Outcome and risk factors of conditional TCD defined as velocities 170–200 cm/sec remains to be described. Patients and methods Since 1992, 371 pediatric SCD patients (303 SS, 44 SC, 18 Sß+, 6 Sß0) were systematically explored once a year by TCD. The newborn screened cohort (n=174) had the first TCD exploration between 12 and 18 months of age. TCD was performed with a real-time imaging unit, using a 2 MHz sector transducer with color Doppler capabilities. Biological data were assessed at baseline, after the age of 1.5 years and remotely of transfusion or VOC. We report the characteristics and the outcome in patients (n=43) with an history of conditional TCD defined by mean maximum velocities ranging between 170 and 200 cm/s in the ACM, the ACA or the ICA. Results: The mean follow-up of TCD monitoring was 5,5 years (0 – 11,8 y). All patients with an history of conditional doppler were SS/Sb0 (n=43). Mean (SD) age of patients at the time of their first conditional TCD was 4.3 years (2.2) whereas in our series the mean age at abnormal TCD (> 200 cm/sec) occurrence was 6.6 years (3.2). Comparison of basal parameters showed highly significant differences between patients with conditional TCD and those with normal TCD: Hb 7g4 vs 8g5 (p<0.001), MCV 82.8 vs 79 (p=0.047). We also had found such differences between patients with normal and those with abnormal TCD (Hb and MCV p< 0.001). Two patients were lost of follow-up. Two patients died during a trip to Africa. Conditional TCD became abnormal in 11/43 patients and justified transfusion program. Mean (SD) conversion delay was 1.8 (2.0) years (range 0.5–7y). No stroke occurred. 16 patients required a treatment intensification for other indications (frequent VOC/ACS, splenic sequestrations): 6 were transplanted and 10 received HU or TP. Significant risk factors (Pearson) of conversion to abnormal were the age at time of conditional TCD occurrence < 3 y (p<0.001), baseline Hb < 7g/dl (p=0.02) and MCV > 80 (p=0.04). MRI/MRA was performed in 31/43 patients and showed ischemic lesions in 5 of them at the mean (SD) age of 7.1 y (1.8) (range 4.5–8.9): no significant difference was observed in the occurrence of lesions between the 2 groups. Conclusions This study confirms the importance of age as predictive factor of conditional to abnormal TCD conversion with a risk of 64% when first conditional TCD occured before the age of 3 years. TCD has to be frequently controled during the 5 first years of life.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2561-2561
Author(s):  
Larisa Geskin ◽  
Francine Foss ◽  
Madeleine Duvic ◽  
David Straus ◽  
Steven Horwitz ◽  
...  

Abstract Background: Mycosis Fungoides (MF) and its leukemic variant Sezary syndrome (SS) are disorders of malignant, skin homing helper/memory T-cells. MF presents with patches, plaques, or tumors, while SS presents with generalized erythroderma and blood involvement. Either can involve lymph nodes, blood, and viscera. A multi-center, open label, single arm clinical trial previously demonstrated the safety and efficacy of ECP as a monotherapy in the treatment of patients with advanced/refractory MF/SS (Edelson, et al, 1987, N Engl J Med, 316:297–303). The primary endpoint of this study was a ≥25% improvement in skin score maintained for at least 4 weeks. We present a long-term, secondary analysis of these patients to further evaluate clinical outcomes and predictors of response for ECP as a monotherapy in MF/SS. Partial (≥50%) and complete (≥90%) skin score responses, extent of skin disease, number of ECP treatments administered, and the time required to achieve 50% and 90% improvement in skin involvement were evaluated. Patients and Methods: Thirty-nine patients (pts) who met eligibility criteria were included in the secondary efficacy analysis as the intent-to-treat (ITT) patient population. Thirty-one pts with generalized erythroderma (GE) and 8 pts with extensive patch plaque (EPP) were treated with ECP on 2 consecutive days every 4–5 weeks for 3 months. No concomitant systemic medications for MF/SS were allowed on study; however, topical steroids could be applied to the hands and feet. Patients had received an average of 3.7 (range 0–13) prior therapies (systemic and topical). Immediately prior to undergoing ECP, all pts received oral doses of methoxsalen in order to achieve blood level concentrations ≥50 ng/mL. Skin improvement was calculated by comparing baseline skin score to skin scores on all subsequent treatment dates. The mean baseline skin score of the 39 ITT patients was 262 (median = 291) based on a maximum possible skin score of 400 points. Results: The median follow-up of the 39 ITT pts was approximately 4 years (range 9 days–7.8 years). Twenty-nine pts (74%) achieved at least a ≥50% improvement in skin score, and 16 pts (41%) achieved ≥90% improvement on ECP monotherapy. The type and extent of skin disease (GE vs. EPP) prior to the start of treatment did not predict response. Patients received a median of 12 (range 4–65) or 30.0 (range 12–109) individual ECP treatments to achieve a ≥50% or ≥90% response, respectively. The mean times to reach a ≥50% or ≥90% response were 8.4 ± 6 months (median=6.5) or 25.2 ±14.9 mos (median=19.6), respectively. The mean duration of a ≥50% response was 32.5 ± 28.6 mos, which included a median of 20 (range 0–153) ECP treatments. Median survival from date of diagnosis and from date of first ECP treatment was 10.6 yrs and 5.4 yrs, respectively. Conclusions: In this long-term, follow-up analysis, ECP monotherapy was associated with a significant and durable improvement in skin score in the majority of patients with MF/SS.


2021 ◽  
Vol 3 (Supplement_6) ◽  
pp. vi27-vi27
Author(s):  
Atsufumi Kawamura ◽  
Junji Koyama ◽  
Nobuyuki Akutsu ◽  
Masashi Higashino ◽  
Kenji Fujita

Abstract Optic pathway glioma(OPG) is almost recognized in childhood and about 0.01–0.02% of whole brain tumor in Japan. Because of the rare tumor, there are few reports about results of its treatment. In 2021, Guideline of Optic pathway/hypothalamic glioma is indicated from The japan Society for Neuro-Oncology. We retrospectively study 9 cases history of OPG who have treated for more than 5 years from January 2005 to March 2021 in Kobe Children’s Hospital. Cases are 4 boys and 5 girls. Average age at diagnosis is 3.8 years old and average follow up term is 11 years 10 months. They are 4 Pilocytic astrocytoma, 3 Pilomyxoid astrocytoma, and 1 Fibrillary astrocytoma. Al l cases have survived. There are only 2 cases who could be controlled with single series of surgical treatment and chemotherapy. Most cases need several times of resection and chemotherapy and uncontrollable 5 cases required radiological treatment. 2 cases are still under treatment for over 10 years. For OPG, partial resection to control hydrocephalus is recommended and several trial of chemotherapy must be carried out. There exist a few cases who need continuous treatment for long term. The other side, a few cases are uncontrollable those need radiotherapy to manage tumor volume. Because the history of OPG would be long term, we should adjust the treatment plan to environment of patients.


2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Ashley M. Cooper ◽  
Elaine R. Flanagan ◽  
Tova Ronis ◽  
Baruch Goldberg ◽  
Ashley K. Sherman ◽  
...  

Abstract Background Chronic anterior uveitis is a sight-threatening complication of juvenile idiopathic arthritis (JIA) and a primary contributor to long-term morbidity in people with JIA. Levels of knowledge about uveitis among JIA patients and their parents are unknown. A survey of JIA patients and parents was conducted to assess knowledge about uveitis complications and recommended screening. Methods A survey was developed consisting of six demographic questions, six arthritis/uveitis history questions, and nine uveitis knowledge questions. The survey was administered to JIA patients age 14 and older and parents of patients with JIA at three pediatric rheumatology practices and online through the Patients, Advocates, and Rheumatology Teams Network for Research and Service (PARTNERS) network. ANOVA, chi-square and Fisher’s exact tests were used to look for relationships between survey questions and demographic variables. Results Thirty-three patients and 111 parents completed the survey. Overall, 17.4% reported a history of uveitis, and 89.6% had heard of uveitis. The mean composite knowledge score was 6.46 ± 2.6 out of 9. Patients and parents with a history of uveitis had higher composite knowledge scores than their counterparts without a uveitis history (p = 0.01 and p < 0.01, respectively). Parents whose rheumatologist reminded them about eye exams at every visit had higher knowledge of the risk of blindness (p = 0.04), the risk for uveitis when arthritis is controlled (p = 0.02), the need for ongoing eye exams when off of medications (p = 0.01), and had a higher overall score (p = 0.02) than those who were reminded at some visits or not at all. Conclusions JIA patients and parents report variable levels of knowledge regarding uveitis complications and recommended screening. Frequent discussion between the rheumatology provider and family about uveitis screening is associated with higher uveitis knowledge. Incorporating detailed and frequent education about uveitis into rheumatology clinic appointments may improve early uveitis detection and visual outcomes.


2018 ◽  
Vol 90 (2) ◽  
pp. 136 ◽  
Author(s):  
Cem Yücel ◽  
Salih Budak ◽  
Mehmet Zeynel Keskin ◽  
Erdem Kisa ◽  
Zafer Kozacioglu

Objective: To observe the clinical practice of salvage microdissection testicular sperm extraction (mTESE) in patients with non-obstructive azoospermia (NOA) and to determine the factors that may predict the presence of spermatozoa in preoperative salvage mTESE. Methods: We retrospectively reviewed the medical records of 445 patients with the diagnosis of NOA, who had undergone the mTESE operation consecutively in our institution between the dates of March 2008 and June 2017. The study included a total of 49 patients with failure to detect spermatozoa in the first mTESE and who had then undergone salvage mTESE. In order to investigate the factors that predict the result of salvage mTESE, the patients were classified into two groups according to the outcome of salvage mTESE, as those with and without spermatozoa retrieval. Patients in these two groups were compared with regard to age, body mass index, history of varicocele, history of cryptorchidism, duration of infertility, outcomes of genetic analysis, results of hormone profiles and the testicular histopathology results of the first mTESE. Results: The sperm retrieval rate following salvage mTESE was observed to be 42.8%. Statistically a significant difference was determined between the mean follicle stimulating hormone (FSH) values of the groups (p = 0.013). No significant difference was observed between the groups with regard to the remaining parameters.Conclusion: It was observed that among the factors that predict the success of sperm retrieval in salvage mTESE in patients with NOA and previous unsuccessful sperm retrieval in mTESE operation, only the pre-operative FSH level was observed to significantly correlate with the success in salvage mTESE.


2014 ◽  
Vol 2014 ◽  
pp. 1-5 ◽  
Author(s):  
Peri Arbak ◽  
İlknur Başer ◽  
Özlem Ozdemir Kumbasar ◽  
Füsun Ülger ◽  
Zeki Kılıçaslan ◽  
...  

Aim. This study aimed to assess the long-term respiratory effects of tear gases among the subjects with history of frequent exposure.Materials and Methods. A questionnaire by NIOSH and pulmonary function tests was performed in 93 males exposed to the tear gases frequently and 55 nonexposed subjects.Results. The mean numbers of total exposure and last 2 years exposure were8.4±6.4times,5.6±5.8times, respectively. Tear gas exposed subjects were presented with a higher rate for cough and phlegm more than 3 months (24.7% versus 11.3%,P>0.05). Mean FEV1/FVC and % predicted MMFR in smoker exposed subjects are significantly lower than those in smoker controls (81.7% versus 84.1%,P=0.046and 89.9% versus 109.6%,P=0.0004, resp.). % predicted MMFR in nonsmoker exposed subjects is significantly lower than that in nonsmoker controls (99.4% versus 113.1%,P=0.05). Odds ratios for chest tightness, exercise dyspnea, dyspnea on level ground, winter morning cough, phlegm, and daily phlegm were increased almost 2 to 2.5 folds among tear gas exposed subjects.Conclusion. The rates for respiratory complaints were high in the case of the exposure to the tear gases previously. Tears gas exposed subjects were found to be under the risk for chronic bronchitis.


2017 ◽  
Vol 1 (2) ◽  
Author(s):  
Yu Hu ◽  
Lin Lin ◽  
Pangen Cui ◽  
Xu Yao ◽  
Chao Luan ◽  
...  

Severe psoriasis patients are reported to have a higher risk of liver abnormalities. Treatment option for severe psoriasis patients with liver disorder history remains a great challenge. Hepatic toxicity and long-term safety are the major concerns. Hence it is necessary to look for safer and more effective treatment for those patients. This retrospective review evaluated safety and efficacy of combination therapy of infliximab and TGP in treating 13 severe psoriasis patients with liver disorder history.Thirteen patients with severe psoriasis, including 8 men and 5 women, with a mean age of 37.3 ± 12.3 were observed. The patients experienced a mean course of psoriasis of 11.2 ± 7.1 years. The mean PASI score was 29.3 ± 12.9. All patients have the history of liver disorder. In our study, these patients were treated with infliximab at a dose of 5mg/kg and TGP at a dose of 1.8g/day. No liver test abnormalities were seen during combination therapy. After treatment, 61.5% patients showed PASI 50 response at week 2, and 81.8% patients have PASI 75 response at week 6. The mean time for achieving PASI 75 and PASI 90 improvement was 4.2 week and 9.6 week, respectively. Conclusion: Our observation demonstrates that combined therapy of infliximab and TGP is effective and safe in the treatment of severe psoriasis, especially for the patients with liver disorder history.


F1000Research ◽  
2021 ◽  
Vol 10 ◽  
pp. 165
Author(s):  
Supawan Surukrattanaskul ◽  
Pukkapol Suvannachart ◽  
Sunee Chansangpetch ◽  
Anita Manassakorn ◽  
Visanee Tantisevi ◽  
...  

Purpose: To evaluate the clinical characteristics and treatment outcomes of patients with childhood glaucoma. Methods: We retrospectively reviewed the data of patients with childhood glaucoma who visited the glaucoma clinics at the Queen Sirikit National Institute of Child Health and the King Chulalongkorn Memorial Hospital between January 2008 and January 2018. The diagnosis was based on the Childhood Glaucoma Research Network classification. We recorded their clinical characteristics and requirement of any glaucoma interventions. Results: A total of 691 eyes from 423 patients were included in this study. The patients predominantly comprised boys. The average follow-up duration was 71.3±63.8 months. The mean age at presentation was 3.9±4.4 years. Most patients presented with a high initial intraocular pressure (IOP) of 28.5±11.2 mmHg. Glaucoma associated with non-acquired ocular anomalies (22.9%) was the most common subtype, followed by primary congenital glaucoma (20.8%). We recorded a family history of glaucoma in 6.4% of patients. Most patients had bilateral glaucoma (63.4%) and required at least one intervention (51.5%). The average IOP at the latest follow-up visit was 19.1±10.8 mmHg. All glaucoma types had significantly lower IOP, compared to that at their baselines (all p<0.001). Moreover, most patients had an unfavourable visual acuity (49.5%) at their latest visit. Conclusions: Secondary glaucoma associated with non-acquired ocular anomalies is the most common subtype of glaucoma. All subtypes, including primary glaucoma, were sporadic. The majority of patients had unfavourable visual outcomes. These real-world findings are fundamental to acquire a better understanding of childhood glaucoma.


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