scholarly journals Safety and Effectiveness of Oral Methylprednisolone Therapy in Comparison With Intramuscular Adrenocorticotropic Hormone and Oral Prednisolone in Children With Infantile Spasms

2021 ◽  
Vol 12 ◽  
Author(s):  
Hong-Min Zhu ◽  
Chun-Hui Yuan ◽  
Meng-Qing Luo ◽  
Xiao-Long Deng ◽  
Sheng Huang ◽  
...  
Keyword(s):  
Adverse Effects ◽  
Adrenocorticotropic Hormone ◽  
Oral Prednisolone ◽  
Infantile Spasms ◽  
Newly Diagnosed ◽  
First Line ◽  
Short Term ◽  
Open Label ◽  
Recurrence Rates ◽  
Electroencephalogram Eeg

Background and Purpose: To assess the safety and effectiveness of oral methylprednisolone (oMP) in comparison with intramuscular adrenocorticotropic hormone (imACTH) and oral prednisolone (oP) therapies in children with infantile spasms (IS).Methods: In this prospective, open-label, non-blinded, uncontrolled observational study, children (aged 2–24 months) with newly diagnosed IS presenting with hypsarrhythmia or its variants on electroencephalogram (EEG) were included. It was followed by imACTH, oP, or oMP (32–48 mg/day for 2 weeks followed by tapering) treatments. Electroclinical remission/spasm control, relapse, and adverse effects were evaluated in the short-term (days 14 and 42) and intermediary-term (3, 6, and 12 months) intervals.Results: A total of 320 pediatric patients were enrolled: 108, 107, and 105 in the imACTH, oMP, and oP groups, respectively. The proportion of children achieving electroclinical remission on days 14 and 42 was similar among the three groups (day 14: 53.70 vs. 60.75 vs. 51.43%, p = 0.362; day 42: 57.55 vs. 63.46 vs. 55.34%, p = 0.470). The time to response was significantly faster in the oMP group (6.5 [3.00, 10.00] days vs. 8.00 [5.00, 11.00] days for imACTH and 8.00 [5.00, 13.00] days for oP, p = 0.025). Spasm control at 3, 6, and 12 months was also similar in the three groups (P = 0.775, 0.667, and 0.779). The relapse rate in the imACTH group (24.10%) was lower than oMP (30.77%) and oP groups (33.33%), and the time taken for relapse in the imACTH group (79.00 [56.50, 152.00] days) was longer than oMP (62.50 [38.00, 121.75] days) and oP groups (71.50 [40.00, 99.75] days), but the differences were not statistically significant (p = 0.539 and 0.530, respectively). The occurrence of adverse effects was similar among the three groups.Conclusions: The short and intermediary-term efficacy and recurrence rates of oMP are not inferior to those of imACTH and oP for the treatment of IS. Significantly, the time to achieve electroclinical remission with oMP was quicker than that with imACTH and oP. Considering its convenience, affordability, and the absence of irreversible side effects, oMP can serve as a form of first-line treatment for newly diagnosed IS.

scholarly journals Short-term outcomes associated with temozolomide vs. PCV chemotherapy for 1p/19q-codeleted WHO grade III anaplastic oligodendrogliomas: a national evaluation

2020 ◽  
Author(s):  
Nayan Lamba ◽  
Malia McAvoy ◽  
Vasileios K. Kavouridis ◽  
Timothy R. Smith ◽  
Mehdi Touat ◽  
...  
Keyword(s):  
Cox Regression ◽  
Long Term Results ◽  
National Database ◽  
Newly Diagnosed ◽  
First Line ◽  
Short Term ◽  
Who Grade ◽  
Cox Regression Analysis ◽  
Pcv Chemotherapy ◽  
Grade Iii

AbstractPURPOSEThe optimal chemotherapy regimen between temozolomide (TMZ) and procarbazine, lomustine, and vincristine (PCV) remains uncertain for newly-diagnosed anaplastic oligodendroglioma (AO). We therefore addressed this question using a national database.METHODSPatients newly-diagnosed with 1p/19q-codeleted W.H.O. grade III AO between 2010-2016 were identified from the National Cancer Database. Predictors of receiving first-line single-agent TMZ vs. multi-agent PCV were assessed by multivariable logistic regression. Overall survival (OS) was estimated by Kaplan-Meier techniques and evaluated by multivariable Cox regression.RESULTS1,360 AO patients were identified: 74.5% (n=1,013) treated with TMZ, 9.6% (n=131) with PCV, and 15.9% (n=216) with no chemotherapy in the first-line setting. In multivariable logistic analysis, PCV utilization increased from 2010 to 2016 (OR=1.38/year, 95%CI: 1.22-1.56, p<0.001) and was less commonly utilized in privately insured patients (OR=0.38 vs. uninsured, 95%CI: 0.15-0.97, p=0.04). In survival analyses (33.1% reached endpoint), there was no difference in unadjusted OS between TMZ (5yr-OS 60.1%, 95%CI: 55.9-64.1) and PCV (5yr-OS 61.1%, 95%CI: 45.6-73.5; p=0.42). There remained no OS difference between TMZ and PCV in the 75.9% (n=1,032) of AO patients that also received radiotherapy (p=0.51), in the Cox regression analysis adjusted by age, extent of resection, and radiotherapy (TMZ vs. PCV HR=1.31, 95%CI: 0.83-2.08, p=0.24), and in subgroup analyses that incorporate KPS or MGMT status.CONCLUSIONSIn a national database of AOs managed in the ‘real-world’ setting, there is no difference in the short-term mortality between first-line TMZ and PCV chemotherapy. These findings provide preliminary data while we await the long-term results from the CODEL trial.

scholarly journals Infantile Spasms in Children With Down Syndrome: Identification and Treatment Response

2019 ◽  
Vol 6 ◽  
pp. 2333794X1882193 ◽  
Author(s):  
Dee Daniels ◽  
Kelly Knupp ◽  
Tim Benke ◽  
Kristine Wolter-Warmerdam ◽  
Maura Moran ◽  
...  
Keyword(s):  
Down Syndrome ◽  
Adrenocorticotropic Hormone ◽  
Early Recognition ◽  
Heart Defects ◽  
Hormone Treatment ◽  
Magnetic Resonance Images ◽  
Infantile Spasms ◽  
Single Institution ◽  
First Line ◽  
Children With Down Syndrome

Objectives. To evaluate infantile spasms in children with Down syndrome including assessment of efficacy of treatments, presence of treatment lag, and to identify risk factors that may predict the occurrence of infantile spasms in this population. Methods. Medical charts, electroencephalograms, and brain magnetic resonance images were evaluated in 37 children treated for infantile spasms at a single institution from 2005 to 2015. Results. Mean age at diagnosis was 9.16 months, with an average 1.38-month lag from spasms onset to start of medication. Prevalence of heart defects and pulmonary hypertension were significantly higher in those with infantile spams compared with those without. Eighty-one percent receiving adrenocorticotropic hormone as initial treatment experienced remission within 2 weeks, 94.1% had remission at 3 months compared with 18.8% at 2 weeks and 35.3% at 3 months for other first-line treatments. Type of treatment was the only predictor of good outcome. Conclusions. Results stress the importance of early recognition and adrenocorticotropic hormone treatment for this seizure disorder in children with Down syndrome.

scholarly journals Lamotrigine versus Valproic Acid as First-line Monotherapy in Newly Diagnosed Typical Absence Seizures: An Open-label, Randomized, Parallel-group Study

Epilepsia ◽  
2004 ◽  
Vol 45 (9) ◽  
pp. 1049-1053 ◽  
Author(s):  
Giangennaro Coppola ◽  
Gianfranca Auricchio ◽  
Rosario Federico ◽  
Marco Carotenuto ◽  
Antonio Pascotto
Keyword(s):  
Valproic Acid ◽  
Newly Diagnosed ◽  
First Line ◽  
Open Label ◽  
Absence Seizures ◽  
Parallel Group Study ◽  
Parallel Group

scholarly journals ACTR-23. MAINTENANCE TREATMENT WITH IBRUTINIB FOLLOWING FIRST LINE METHOTREXATE-BASED IMMUNO-CHEMOTHERAPY IN ELDERLY PATIENTS WITH PRIMARY CNS LYMPHOMA (PCNSL). A PHASE 2 OPEN-LABEL STUDY

2019 ◽  
Vol 21 (Supplement_6) ◽  
pp. vi17-vi18
Author(s):  
Osnat Bairey ◽  
Alexandra Benouaich-amiel ◽  
Shlomit Yust-Katz ◽  
Ronit Gurion ◽  
Tali Siegal
Keyword(s):  
Maintenance Treatment ◽  
Fungal Infections ◽  
Randomized Study ◽  
Primary Cns Lymphoma ◽  
Progression Free Survival ◽  
Cns Lymphoma ◽  
High Dose ◽  
Newly Diagnosed ◽  
First Line ◽  
Open Label

Abstract BACKGROUND Patients older than 60 years account for up to 70% of all PCNSL cases. Elderly PCNSL patients have median overall survival (OS) under 2 years and progression free survival (PFS) ranging between 6–16 months. Older patients have multiple comorbidities associated with low tolerability to high-dose (HD) chemotherapy. As maintenance treatment prolongs PFS and\or OS in several hematological malignancies we sought to investigate whether Ibrutinib maintenance may benefit elderly PCNSL patients. Ibrutinib was selected for maintenance since it has an impressive tolerability and activity in a range of systemic B-cell lymphomas. METHODS Single arm, open label, non-randomized study aiming to accrue 30 newly diagnosed PCNSL patients aged 60–85 years who received HD-methotrexate–based first line chemotherapy and have a documented response which is either partial (PR) or complete response (CR). The primary end-point is one and 2-year PFS and ibrutinib dose is 560mg/day. All patients undergo pre-maintenance neurocognitive evaluation which is repeated every 6 months. RESULTS Of the 16 patients screened for the study 2 were excluded due to relapse while on screening. 14 patients have been enrolled with a median age of 74 (61–80) years. The median interval between PCNSL diagnosis and start of ibrutinib maintenance is 7.6 (5.6–11.5) months. Currently, the median PFS is 22.5 (12–31.5) months. The adverse effects are largely grade 1/2 with rare grade 3/4 events. One patient discontinued treatment due to skin rash at 4.5 months. Two patients relapsed while on maintenance after 4 and 15 months of treatment. 3 patients with PR at enrolment improved to CR/CRu during maintenance. No invasive fungal infections have been observed. CONCLUSIONS Ibrutinib maintenance is feasible and well tolerated in newly diagnosed elderly PCNSL patients after first-line HD-MTX based treatment. The toxicity is mild to moderate. Enrollment is ongoing and updated outcomes will be presented at the meeting.

First-line rituximab combined with short-term prednisone versus prednisone alone for the treatment of pemphigus (Ritux 3): a prospective, multicentre, parallel-group, open-label randomised trial

The Lancet ◽  
2017 ◽  
Vol 389 (10083) ◽  
pp. 2031-2040 ◽  
Author(s):  
Pascal Joly ◽  
Maud Maho-Vaillant ◽  
Catherine Prost-Squarcioni ◽  
Vivien Hebert ◽  
Estelle Houivet ◽  
...  
Keyword(s):  
Randomised Trial ◽  
First Line ◽  
Short Term ◽  
Open Label ◽  
Parallel Group

scholarly journals Immunotherapies for Anti-NMDA Receptor Encephalitis: Multicenter Retrospective Pediatric Cohort Study in China

2021 ◽  
Author(s):  
Shiqi Guang ◽  
Jiannan Ma ◽  
Xiaotun Ren ◽  
Shuizhen Zhou ◽  
Jian Yang ◽  
...  
Keyword(s):  
Relapse Rate ◽  
Pediatric Patients ◽  
Oral Prednisolone ◽  
Second Line ◽  
First Line ◽  
Short Term ◽  
Treatment Groups ◽  
Nmdar Encephalitis ◽  
Clinical Records

Abstract BackgroundAnti-N-methyl-D-aspartate receptor (NMDAR) encephalitis has been discovered and termed more than a decade, but the establishment of standardized immunotherapy protocol for pediatric patients still needs more clinical evidence. To help move this forward, we investigated the current status of immunotherapies for pediatric anti-NMDAR encephalitis in 6 tertiary medical centers across China and evaluated how different immunotherapy strategies affected patient outcomes.MethodsA multicenter, retrospective study was conducted on pediatric patients diagnosed with anti-NMDAR encephalitis between November 2011 and December 2018. The clinical records including clinical manifestations, immunotherapy strategies, and outcomes were collected and analyzed. Treatment response and outcome were evaluated using mRS. Outcomes among the treatment groups were analyzed with the Chi-squared test or Fisher’s exact test. p < 0.05 was considered significant.ResultsA total of 386 patients were included in our study and the median onset age was 7.89 (range 0.5-18) years. All patients received first-line immunotherapy and the majority (341, 88.3%) used the standard combination of methylprednisolone pulses and intravenous immunoglobulins, but 211 patients did not show satisfactory improvement (mRS ≥ 3). Mainly three treatment strategies were applied after first-line immunotherapy: second-line immunotherapy, repetitive first-line immunotherapy, and maintaining oral prednisolone. For patients with mRS ≥ 4 after first-line immunotherapy, the incidence of poor outcome (mRS ≥ 3) in oral prednisolone group was higher than that in other treatment groups (0.025 < p < 0.05). No difference in complete recovery rate (mRS = 0) was found between patients receiving second-line and repetitive first-line immunotherapy, or patients using long-term and short-term prednisolone. The relapse rate of oral prednisolone group was higher than that of other treatment groups (p < 0.01), but the relapse rate of patients using long-term and short-term prednisolone had no statistical difference. ConclusionsFor patients with mRS ≥ 4 after first-line immunotherapy, second-line immunotherapy is recommended. When second-line immunotherapy is not applicable, repetitive first-line immunotherapy can be considered as an option. Both second-line and repetitive first-line immunotherapy are beneficial to reduce relapse rate. The duration of sequential oral prednisolone can be shortened after fully evaluating patients’ conditions.

scholarly journals Vigabatrin-Induced Encephalopathy in a 5.5-Month-Old Girl with Infantile Spasms due to Tuberous Sclerosis

2019 ◽  
Vol 2019 ◽  
pp. 1-3 ◽  
Author(s):  
Eleni Klinaki ◽  
Ioanna Argyri ◽  
Georgia Amountza ◽  
Gerina Ioannidou ◽  
Despoina Maritsi ◽  
...  
Keyword(s):  
Tuberous Sclerosis ◽  
Tuberous Sclerosis Complex ◽  
Adrenocorticotropic Hormone ◽  
Infantile Spasms ◽  
High Index ◽  
Female Infant ◽  
Eeg Abnormalities ◽  
High Index Of Suspicion ◽  
Electroencephalogram Eeg

A 5.5-month-old female infant with tuberous sclerosis complex presented with infantile spasms and was treated with vigabatrin. As her condition did not improve, she was given adrenocorticotropic hormone (ACTH) intramuscularly which stopped the spasms and improved the electroencephalogram (EEG) abnormalities. However, she developed encephalopathy with apathy, drowsiness, and generalized slowing in the EEG. Discontinuation of vigabatrin quickly improved her symptoms and reversed the EEG slowing. A high index of suspicion is required in order to diagnose vigabatrin-induced encephalopathy, especially as the underlying disorders of these patients can be erroneously considered the cause of the observed encephalopathy.

scholarly journals P05.04 Phase 2 open-label study of maintenance treatment with ibrutinib following first line methotrexate-based immuno-chemotherapy in elderly patients with primary CNS lymphoma (PCNSL)

2019 ◽  
Vol 21 (Supplement_3) ◽  
pp. iii34-iii34
Author(s):  
O Bairey ◽  
A Amiel ◽  
S Yust-Katz ◽  
R Gurion ◽  
T Siegal
Keyword(s):  
Maintenance Treatment ◽  
Fungal Infections ◽  
Randomized Study ◽  
Primary Cns Lymphoma ◽  
Progression Free Survival ◽  
Cns Lymphoma ◽  
High Dose ◽  
Newly Diagnosed ◽  
First Line ◽  
Open Label

Abstract BACKGROUND Patients older than 60 years account for up to 70% of all PCNSL cases. Elderly PCNSL patients have median overall survival (OS) under 2 years and progression free survival (PFS) ranging between 6–16 months. Older patients have multiple comorbidities associated with low tolerability to high-dose (HD) chemotherapy. As maintenance treatment proved to prolong PFS and\or OS in several hematological malignancies we sought to investigate whether Ibrutinib maintenance may benefit elderly PCNSL patients. Ibrutinib was selected for maintenance since it has an impressive tolerability and activity in a range of systemic B-cell lymphomas. MATERIAL AND METHODS Single arm, open label, non-randomized study aiming to accrue 30 newly diagnosed PCNSL patients aged 60–85 years who received HD-methotrexate-based first line chemotherapy and have a documented response which is either partial (PR) or complete response (CR). The primary end-point is one and 2-year PFS and maintenance ibrutinib dose is 560mg/day. All patients undergo pre-maintenance neurocognitive evaluation which is repeated every 6 months. RESULTS Of the 14 patients screened for the study 2 were excluded due to relapse while on screening. 12 patients have been enrolled with a median age of 74 (61–80) years. The median interval between diagnosis of PCNSL and start of ibrutinib maintenance is 7.6 (5.6–11.5) months. Currently, the median PFS is 22.5 (12–31.5) months. The adverse effects are largely grade 1/2 with rare grade 3/4 events. One patient discontinued treatment due to skin rash at 4.5 months and she is still alive after 24 months. Two patients relapsed while on maintenance after 4 and 15 months of treatment. 3 patients with PR at enrolment improved to CR/CRu during maintenance. No invasive fungal infections have been observed. CONCLUSION Ibrutinib maintenance is feasible and well tolerated in newly diagnosed elderly PCNSL patients after first-line HD-Methotrexate based treatment. The toxicity is mild to moderate. Enrollment is ongoing and updated outcomes will be presented at the meeting.

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