Complications associated with intrathecal drug delivery in a paediatric patient with Niemann-Pick type C

2021 ◽  
Vol 14 (5) ◽  
pp. e241786
Author(s):  
Jose Ignacio Rodriguez Rodriguez Ciancio ◽  
Kristian Aquilina

We report on a male subject with a diagnosis of Niemann-Pick type C (NPC). He received an experimental medicinal product intrathecally initially via lumbar puncture (LP) and eventually via intrathecal drug delivery device. Shortly after implantation, the device catheter migrated outside of the intrathecal space and coiled subcutaneously. The treatment continued via LP after removal of the device. A subdural haematoma developed after repeated LPs. It was surgically evacuated and the patient recovered with sequelae. Surgically implanted drug delivery devices are designed to bypass the blood–brain barrier and deliver a medicinal product directly into the cerebrospinal fluid circulation. Their use has extended into the field of neurodegenerative disorders. Significant adverse events can occur at any given time after implantation including neurological injury, dislodgement or displacement of any of its components, infection and drug-related complications; all can significantly affect the quality of life of patients. Repeated LPs also carry significant risk.

F1000Research ◽  
2013 ◽  
Vol 2 ◽  
pp. 96 ◽  
Author(s):  
Gaurav Bhatia ◽  
Mary E Lau ◽  
Padma Gulur

Intrathecal drug delivery is an effective pain management option for patients with chronic and cancer pain. The delivery of drugs into the intrathecal space provides superior analgesia with smaller doses of analgesics to minimize side effects while significantly improving quality of life. This article aims to provide a general overview of the use of intrathecal drug delivery to manage pain, dosing recommendations, potential risks and complications, and growing trends in the field.


F1000Research ◽  
2014 ◽  
Vol 2 ◽  
pp. 96 ◽  
Author(s):  
Gaurav Bhatia ◽  
Mary E Lau ◽  
Katharine M Koury ◽  
Padma Gulur

Intrathecal drug delivery is an effective pain management option for patients with chronic and cancer pain. The delivery of drugs into the intrathecal space provides superior analgesia with smaller doses of analgesics to minimize side effects while significantly improving quality of life. This article aims to provide a general overview of the use of intrathecal drug delivery to manage pain, dosing recommendations, potential risks and complications, and growing trends in the field.


2011 ◽  
Vol 14 (2) ◽  
pp. 179-182 ◽  
Author(s):  
Elsa Magro ◽  
Olivier Remy-Neris ◽  
Romuald Seizeur ◽  
Vincent Allano ◽  
Bertrand Quinio ◽  
...  

2021 ◽  
pp. E583-E594

BACKGROUND: Pancreatic cancer (PC) is one of the most lethal cancers and is the eleventh most common cancer worldwide. This disease is characterized by an often-fatal evolution and a high burden of symptoms, particularly pain. Several studies have demonstrated that pancreatic cancer patients have a high prevalence of pain, with up to 82% of patients reporting pain, often requiring systemic strong opioids as mainstay treatment. This comprehensive review of pancreatic cancer related pain (PCRP), focuses on current mechanisms that lead to pain including regional invasion processes, as well as the local secretion of factors that sensitize nociceptive nerves. OBJECTIVE: Our objective was to conduct a review of PCRP and provide updates on intrathecal drug delivery in PC therapeutic recommendations. STUDY DESIGN: We used a narrative review design. We present a novel perspective in the field of pain research by converging data from intrathecal drug delivery trials with previous elements of molecular pain research in PCRP. METHODS: The literature review relating to PCRP pathophysiology and intrathecal drug delivery systems (IDDS) was done with searches of English, French, and Spanish abstracts, using PubMed, Dynamed, EMBASE, SciELO, Uptodate, Google Scholar, and manual searches of the bibliographies of known primary and review articles from IDDS inception until August 2020. Different search strings based on MESH terms were used including: pain, chronic pain, cancer pain, prevalence, pathophysiology, pancreatic cancer, analgesia, invasive pain procedures, celiac plexus neurolysis, pancreatic neuropathy, intrathecal drug delivery, or a combination of these terms. A narrative review based on these sources was prepared. RESULTS: This paper reviews aspects related to pancreatic adenocarcinoma and PCRP prevalence and focuses on recent developments in pathophysiology with IDDS as a pain management strategy. We summarize the best available evidence regarding intrathecal therapy (IT) for PCRP management; 18 studies of IDDS including at least 236 PC patients are analyzed. LIMITATIONS: Some limitations include: IDDS studies heterogeneity regarding disease stage, patient population, and technical aspects, such as catheter placement and treatment regimen, do not allow integration of studies. CONCLUSION: This review analyzes both past and current literature with a critical analysis of findings and respective recommendations. Most studies of IDDS in PCRP evaluate outcomes on pain using one-dimensional pain scales, such as VAS. Other relevant results, such as performance status or quality of life, are not frequently reported. Burden of disease variables, such as cancer stage, location, and comorbidities, like depression and systemic analgesia co-prescription, are usually not presented in these studies. In the same way, most studies do not precisely inform IDDS titration and IT medication. These factors make integration of IDDS in PC studies difficult. Future studies regarding impact of IDDS on pain control on quality of life, in this particular population, may help clinicians in deciding the optimal time and approach for IDDS. The studies should report data on particular disease, comorbidities, and treatment regimens. KEY WORDS: Adenocarcinoma, cancer pain, pain, pancreatic carcinoma, pancreatic neoplasms, pain management, physiopathology, prevalence


2019 ◽  
Vol 28 (11) ◽  
pp. 3083-3092 ◽  
Author(s):  
Lydia Aston ◽  
Rachel Shaw ◽  
Rebecca Knibb
Keyword(s):  
Type C ◽  

2018 ◽  
Vol 43 (6) ◽  
pp. 654-655 ◽  
Author(s):  
Matthew T. McEwan ◽  
Salim M. Hayek ◽  
Ryan Galica ◽  
Vidya Sundaram ◽  
Elias Veizi

2017 ◽  
Vol 32 (1) ◽  
pp. 287-293
Author(s):  
Nishi Patel ◽  
Melanie Huddart ◽  
Helen Makins ◽  
Theresa Mitchell ◽  
Jane L Gibbins ◽  
...  

Background: Intrathecal drug delivery is known to reduce pain in patients where conventional systemic analgesia has been ineffective or intolerable. However, there is little information regarding the effects of intrathecal drug delivery on quality of life and function in those with advanced, incurable cancer. Aim: Retrospective exploration of the views of bereaved carers regarding the physical and psychosocial effects of external tunnelled intrathecal drug delivery in patients with advanced incurable cancer. Design: Thematic analysis of qualitative interviews with carers of deceased individuals who received percutaneous external tunnelled intrathecal drug delivery as part of their pain management, within two UK centres. Setting: A total of 11 carers were recruited from two UK Palliative Care centres. Family carers of adult patients who had received external tunnelled intrathecal drug delivery analgesia for cancer pain and had died between 6 and 48 months prior to contact were included. Carer relatives who were considered likely to be too vulnerable or who had lodged a complaint about treatment within the recruiting department or who had been treated directly by the interviewer were excluded. Results: In total, 11 interviews took place. The emerging themes were (1) making the decision to have the intrathecal – relatives described desperate situations with severe pain and/or sedation, meaning that the individual would try anything; (2) timing and knowing they were having the best – an increased access to pain and palliative care services, meant carers felt everything possible was being done, making the situation more bearable; (3) was it worth it? – the success of the external tunnelled intrathecal drug delivery was judged on its ability to enable the individual to be themselves through their final illness. Side effects were often considered acceptable, if the external tunnelled intrathecal drug delivery enabled improvements in quality of life. Conclusion: Carers perceived external tunnelled intrathecal drug delivery as most valuable when it improved quality of life towards the end of life, by reducing pain and side effects of conventional systemic analgesia to enable individuals ‘to be themselves’. Under these circumstances, the carers judged significant side effects to be acceptable.


2021 ◽  
Vol 22 (12) ◽  
pp. 6600
Author(s):  
Ida Cariati ◽  
Laura Masuelli ◽  
Roberto Bei ◽  
Virginia Tancredi ◽  
Claudio Frank ◽  
...  

Niemann–Pick type C (NPC) disease is an autosomal recessive storage disorder, characterized by abnormal sequestration of unesterified cholesterol in the late endo-lysosomal system of cells. Progressive neurological deterioration and the onset of symptoms, such as ataxia, seizures, cognitive decline, and severe dementia, are pathognomonic features of the disease. In addition, different pathological similarities, including degeneration of hippocampal and cortical neurons, hyperphosphorylated tau, and neurofibrillary tangle formation, have been identified between NPC disease and other neurodegenerative pathologies. However, the underlying pathophysiological mechanisms are not yet well understood, and even a real cure to counteract neurodegeneration has not been identified. Therefore, the combination of current pharmacological therapies, represented by miglustat and cyclodextrin, and non-pharmacological approaches, such as physical exercise and appropriate diet, could represent a strategy to improve the quality of life of NPC patients. Based on this evidence, in our review we focused on the neurodegenerative aspects of NPC disease, summarizing the current knowledge on the molecular and biochemical mechanisms responsible for cognitive impairment, and suggesting physical exercise and nutritional treatments as additional non-pharmacologic approaches to reduce the progression and neurodegenerative course of NPC disease.


2017 ◽  
Vol 8 (4) ◽  
pp. 78-80 ◽  
Author(s):  
Sephalie Patel ◽  
Osama Hafez ◽  
Wade J. Sexton ◽  
David A. Edwards

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