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1.
J Anaesthesiol Clin Pharmacol ; 40(2): 192-198, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38919417

RESUMO

Spinal and epidural blocks are commonly employed for pain relief during and following cesarean section. Intrathecal morphine (ITM) has been the gold standard for the same for many years. In recent times, many peripheral nerve blocks (PNBs) have been tried for postoperative analgesia following cesarean delivery (PACD). This article has reviewed the common PNBs used for PACD. The role of PNBs along with ITM has been studied and the current best strategy for PACD has also been explored. Currently, Ilio-inguinal nerve and anterior transversus abdominis plane block in conjunction with intrathecal morphine have been found to be the most effective strategy, providing lower rest pain at 6 hours as compared to ITM alone. In patients not receiving intrathecal morphine, recommended PNBs are lateral transversus abdominis plane block, single shot local anesthetic wound infiltration, or continuous wound infiltration with catheter below rectus fascia. PNBs are recommended for PACD. They have an opioid-sparing effect and are devoid of adverse effects associated with central neuraxial blocks such as hypotension, bradycardia, and urine retention. However, caution must be observed with PNBs for possible local anesthetic toxicity due to the large volumes of drug required.

2.
J Neurosurg ; 140(1): 291-298, 2024 Jan 01.
Artigo em Inglês | MEDLINE | ID: mdl-37548564

RESUMO

OBJECTIVE: Training of international medical graduates (IMGs) offers opportunities for the US neurosurgery community to engage the global talent pool and impact national and international healthcare. Here, the authors analyzed the time trend of IMGs matching into US neurosurgery programs and identified potential opportunities for enhancing IMG engagement. METHODS: The authors analyzed the National Resident Matching Program (NRMP) match results, NRMP program director (PD) surveys, and applicant surveys from 2013 to 2022. Regression methods were used to analyze time trends. RESULTS: Between 2013 and 2022, the number of US neurosurgery residency positions increased by 17.6% (from 204 to 240). During this period, the percentage of IMGs matching into neurosurgery increased from 3.5% to 7%, translating into a 6.8% increase in the likelihood of a successful IMG match per year (95% CI 0.3%-13.8%, p = 0.042). The likelihoods of a successful match for US MDs and IMGs scoring > 260 on the USMLE Step 1 were > 90% and approximately 55%, respectively. In PD surveys, approximately 90% of PDs indicated that they seldom/never interview or rank IMGs. In terms of factors that influenced the PD decision for interviewing/ranking, IMGs are disadvantaged in several categories, including the ability to secure an audition elective/rotation, and proper letters of recommendation, as well as the influence of the culture on the preconceived perception of poor interpersonal skills. CONCLUSIONS: The number of IMGs matching successfully in neurosurgery has increased marginally during the past decade. The authors outline the challenges that IMGs encounter in this process and suggest strategies for considerations of IMG training in NRMP-associated institutions.


Assuntos
Internato e Residência , Neurocirurgia , Humanos , Estados Unidos , Neurocirurgia/educação , Médicos Graduados Estrangeiros , Educação de Pós-Graduação em Medicina , Procedimentos Neurocirúrgicos
3.
Indian J Anaesth ; 67(7): 595-602, 2023 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-37601942

RESUMO

Background and Aims: The interscalene brachial plexus block (ISB) affects the phrenic nerve, resulting in hemi-diaphragmatic paresis (HDP) and, possibly, respiratory distress. Suprascapular nerve block via an anterior approach (SSB-A) is performed more distally at the level of the trunk of the brachial plexus and, thus, may spare the phrenic nerve. This study compares the analgesic efficacy and decline of hemi-diaphragmatic excursion (HDE) following ultrasound (US)-guided SSB-A versus ISB for arthroscopic shoulder surgery. Methods: This study was conducted on 60 adult participants undergoing arthroscopic shoulder surgery under general anaesthesia. Both US-guided SSB-A (n = 30) and ISB (n = 30) were performed with a combination of 10 ml bupivacaine (0.5%) and 4 mg dexamethasone. The primary objective was to compare the duration of analgesia (time to first rescue analgesia), and secondary objectives were to compare 24-h postoperative numerical rating scale (NRS) scores, 24-h morphine consumption and post block change in HDE, and pulmonary function tests (PFTs) between the two groups. For analysing intergroup differences of NRS, HDE and PFT; Pearson's Chi-squared test or Fisher's exact test, unpaired t test, and Mann-Whitney U test were used. For intragroup differences, paired t test was used. A P value <0.05 was considered significant. Results: The duration of analgesia (mean ± Standard Deviation) was similar in two groups (SSB-A = 1,345 ± 182 min, ISB = 1,375 ± 156 min; P = 0.8). The reduction in HDE was significantly greater in the ISB group (44%) than in the SSB-A group (10%). Pulmonary function was better preserved in the SSB-A group. Conclusion: Compared to ISB, SSB-A has a similar analgesic efficacy for arthroscopic shoulder surgeries, but it is superior in preserving diaphragmatic function and pulmonary function.

4.
Cureus ; 15(6): e40950, 2023 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-37503495

RESUMO

INTRODUCTION:  There is a demand for surgical simulation training to be made accessible in low-resource countries. We conducted a pilot workshop at a new state-of-the-art simulation center and evaluated two novel low-cost surgical simulation models in a lower middle-income country (LMIC). METHODS: A hands-on workshop to train local educators about simulation training was held at a new simulation center. Participant surveys were analyzed following the "training-the-trainer" workshop. Low-cost, hybrid-fidelity pericardiocentesis and thoracic cavity simulation training models were created using locally available materials. These models recreated the pertinent anatomy at a cost under 20 US dollars each. The models were used to train 109 postgraduate anesthesiology trainees during two hands-on medical education workshops. Participant surveys were collected and analyzed. RESULTS: Of the local educators who participated in the "training-the-trainer" workshop, 65% "agreed" and 35% "strongly agreed" with the claim that the simulations better prepared the trainees to teach the clinical scenarios. Additionally, 65% of local educators "agreed" and 35% "strongly agreed" that the simulations prepared them to navigate interprofessional care in those scenarios. The low-cost pericardiocentesis simulation was ranked as "good" or "outstanding" by 100% of survey respondents. The low-cost thoracostomy simulation was ranked as "good" or "outstanding" by 64% of survey respondents. Both the pericardiocentesis and thoracostomy simulators were valued for their low-cost design, the recreation of essential anatomy, and immersive design elements. CONCLUSION: Our team successfully implemented novel simulators for skill training in an LMIC by working in close collaboration with local experts, with the advancement of local simulation instruction practices. Collaboration is key to increasing access to surgical simulations, particularly in low- to middle-income countries.

5.
Ann Card Anaesth ; 26(1): 57-62, 2023.
Artigo em Inglês | MEDLINE | ID: mdl-36722589

RESUMO

Background: Quantification of urinary tissue inhibitor of metalloproteinase-2 (TIMP-2) and insulin-like growth factor binding protein (IFGBP-7), which is commercially known as NephroCheck™(NC) test have been suggested as promising tools for the early detection of acute kidney injury (AKI) after cardiac surgery involving cardio-pulmonary bypass (CPB). Objectives: The aim of the present study was to test the hypothesis that single value of postoperative NC test performed at 4 hours after surgery can predict AKI in off-pump coronary artery bypass grafting (OPCABG) surgery. Setting and Design: This prospective single-center study was conducted at the tertiary cardiac center in India from December 2017 to November 2018. Methods: Ninety adult patients of both sex undergoing elective OPCABG were included. Anesthesia was standardized to all patients. Urine samples were collected preoperatively and at 4 hours after surgery for NC test. Urine output, serum creatinine, estimated glomerular filtration rate (eGFR) were also measured. AKI staging was based on kidney disease improving global outcomes (KDIGO) guidelines. Statistical Analysis: To assess the predictability of NC test for the primary endpoint, area under the receiver operating characteristic curve (ROC), was calculated. Results: Thirteen patients developed AKI in the study cohort (14.4%) out of which 7 patients (7.8%) developed stage 2/3 AKI and the remaining stage 1 AKI. Baseline renal parameters were similar between AKI and non-AKI group. The area under curve (AUC) of NC test at 4 hours after surgery was 0.60 [95% confidence interval (CI): 0.42-0.77]. Postoperative NC test performed at 4 hours after surgery did not predict AKI in this study population (P = 0.24). There were no significant differences in duration of mechanical ventilation, length of intensive care stay and hospital stay between the two groups (P > 0.05). Conclusion: NephroCheck™ test performed at 4 hours after surgery did not identify patients at risk for developing AKI following OPCABG surgery.


Assuntos
Injúria Renal Aguda , Ponte de Artéria Coronária sem Circulação Extracorpórea , Urinálise , Adulto , Humanos , Injúria Renal Aguda/diagnóstico , Injúria Renal Aguda/etiologia , Ponte de Artéria Coronária sem Circulação Extracorpórea/efeitos adversos , Rim , Estudos Prospectivos , Inibidor Tecidual de Metaloproteinase-2/urina , Proteínas de Ligação a Fator de Crescimento Semelhante a Insulina/urina
6.
Br J Anaesth ; 128(2): e109-e119, 2022 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-34862001

RESUMO

The paediatric population is disproportionately affected during mass casualty incidents (MCIs). Several unique characteristics of children merit special attention during natural and man-made disasters because of their age, physiology, and vulnerability. Paediatric anaesthesiologists play a critical part of MCI care for this population, yet there is a deficit of publications within the anaesthesia literature addressing paediatric-specific MCI concerns. This narrative review article analyses paediatric MCI considerations and compares differing aspects between care provision in Australia, the UK, and the USA. We integrate some of the potential roles for anaesthesiologists with paediatric experience, which include preparation, command consultation, in-field care, pre-hospital transport duties, and emergency department, operating theatre, and ICU opportunities. Finally, we propose several methods by which anaesthesiologists can improve their contribution to paediatric MCI care through personal education, training, and institutional involvement.


Assuntos
Serviços Médicos de Emergência/organização & administração , Serviço Hospitalar de Emergência/organização & administração , Incidentes com Feridos em Massa , Fatores Etários , Anestesia/métodos , Anestesiologistas/organização & administração , Criança , Humanos , Pediatria
7.
Ann Card Anaesth ; 24(3): 384-388, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34269276

RESUMO

Massive pulmonary hemorrhage during pulmonary thromboendarterectomy (PTE) can be managed by a conservative approach with mechanical ventilatory support, positive end-expiratory pressure, lung isolation, reversal of heparin, and correct of coagulopathy. We present three challenging cases that developed intrapulmonary hemorrhage during/after PTE and managed successfully. The first patient had bleeding from the bronchial artery and right internal mammary collaterals, which was managed by coil-embolization. The second patient had a breach in the blood airway barrier in the right upper lobar segment of the lung, and the repair was done using a surgical absorbable hemostat. The third patient developed reperfusion injury, he was instituted on veno-venous extracorporeal membranous oxygenation, a week later, the patient recovered completely. An algorithm was adopted and modified to our requirements; all the 3 challenging intrapulmonary hemorrhage cases were successfully managed. This algorithm can be used for satisfactory outcomes in patients who suffer intrapulmonary hemorrhage during PTE.


Assuntos
Hipertensão Pulmonar , Embolia Pulmonar , Doença Crônica , Endarterectomia , Hemorragia/etiologia , Hemorragia/terapia , Humanos , Recém-Nascido , Pulmão , Masculino , Artéria Pulmonar , Embolia Pulmonar/complicações , Embolia Pulmonar/diagnóstico por imagem , Embolia Pulmonar/cirurgia
8.
J Med Syst ; 45(2): 21, 2021 Jan 11.
Artigo em Inglês | MEDLINE | ID: mdl-33426609

RESUMO

Technology improvements have rapidly advanced medicine over the last few decades. New approaches are constantly being developed and utilized. Anesthesiology strongly relies on technology for resuscitation, life-support, monitoring, safety, clinical care, and education. This manuscript describes a reverse engineering process to confirm the fit of a medical device in a pediatric patient. The method uses virtual reality and three-dimensional printing technologies to evaluate the feasibility of a complex procedure requiring one-lung isolation and one-lung ventilation. Based on the results of the device fit analysis, the anesthesiology team confidently proceeded with the operation. The approach used and described serves as an example of the advantages available when coupling new technologies to visualize patient anatomy during the procedural planning process.


Assuntos
Anestesiologia , Criança , Computadores , Humanos , Imageamento Tridimensional , Cuidados Pré-Operatórios , Impressão Tridimensional
10.
Ann Card Anaesth ; 23(4): 465-470, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33109805

RESUMO

Background: This study was undertaken to determine the effects of dexmedetomidine on pulmonary artery pressure (PAP) in children with congenital heart disease (CHD) and pulmonary hypertension (PH) undergoing cardiac catheterization with and without a planned intervention during monitored anesthetic care using midazolam and ketamine. Materials and Methods: Children (<18 years) with known CHD and PH who were scheduled for cardiac catheterization and interventional procedures were included in the study. The procedures were performed under monitored anesthesia. After obtaining baseline PAPs, an intravenous (IV) infusion of dexmedetomidine (1 µg/kg) was given for over 10 min. During infusion, heart rate (HR), blood pressure (BP), respiratory rate (RR), and peripheral arterial oxygen saturation (SPO2) were recorded every 2 min until completion of dexmedetomidine infusion, 15 min later, and when the procedure was completed. In addition, pulmonary artery systolic and diastolic pressures, and mean pulmonary artery pressure (MPAP) were recorded and the pulmonary artery systolic pressure (PASP)/systolic blood pressure (BP) ratio was calculated. Results: All children tolerated the procedure without adverse events. The HR decreased significantly over time during dexmedetomidine infusion. The changes in systemic systolic BP and PAPs were not significantly different from the baseline value at all points of measurement as was the ratio between the systolic pulmonary artery and systolic systemic BPs. Conclusions: Administration of dexmedetomidine in a dose of 1 µg/kg over 10 min did not significantly alter the PAP in children with CHD and PH. There was a decrease in the HR that was not clinically significant. The children tolerated dexmedetomidine without adverse events.


Assuntos
Dexmedetomidina , Cardiopatias Congênitas , Hipertensão Pulmonar , Criança , Cardiopatias Congênitas/complicações , Hemodinâmica , Humanos , Hipertensão Pulmonar/tratamento farmacológico , Estudos Prospectivos , Artéria Pulmonar
12.
Anesthesiol Clin ; 38(3): 643-662, 2020 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-32792189

RESUMO

The focus of this article is noncardiac surgery in the adult with congenital heart disease (CHD). The purpose is to provide the general and pediatric anesthesiologist with a basic overview of the most common congenital cardiac lesions, their long-term sequelae, and expected perioperative concerns during noncardiac surgery. Because of the very heterogeneous nature of CHD, it is difficult to make a single article a comprehensive guide for every lesion and its associated perioperative concerns. The authors hope to provide those who are not specifically trained in congenital cardiac anesthesia the basic principles and a greater understanding of each defect.


Assuntos
Anestesia/métodos , Cardiopatias Congênitas/fisiopatologia , Procedimentos Cirúrgicos Operatórios , Adulto , Humanos
13.
Am J Infect Control ; 48(10): 1237-1243, 2020 10.
Artigo em Inglês | MEDLINE | ID: mdl-32603849

RESUMO

BACKGROUND: To mitigate potential exposure of healthcare workers (HCWs) to SARS-CoV-2 via aerosol routes, we have developed a portable hood which not only creates a barrier between HCW and patient, but also utilizes negative pressure with filtration of aerosols by a high-efficiency particulate air filter. MATERIAL AND METHODS: The hood has iris-port openings for access to the patient, and an opening large enough for a patient's head and upper torso. The top of the hood is a high-efficiency particulate air filter connected to a blower to apply negative pressure. We determined the aerosol penetration from outside to inside in laboratory experiments. RESULTS: The penetration of particles from within the hood to the breathing zones of HCWs outside the hood was near 10-4 (0.01%) in the 200-400 nm size range, and near 10-3 (0.1%) for smaller particles. Penetration values for particles in the 500 nm-5 µm range were below 10-2 (1%). Fluorometric analysis of deposited fluorescein particles on the personal protective equipment of an HCW revealed that negative pressure reduces particle deposition both outside and inside the hood. CONCLUSIONS: We find that negative pressure hoods can be effective controls to mitigate aerosol exposure to HCWs, while simultaneously allowing access to patients.


Assuntos
Infecções por Coronavirus/prevenção & controle , Filtração/instrumentação , Transmissão de Doença Infecciosa do Paciente para o Profissional/prevenção & controle , Pandemias/prevenção & controle , Equipamento de Proteção Individual , Pneumonia Viral/prevenção & controle , Infecções Respiratórias/prevenção & controle , Adulto , Betacoronavirus , COVID-19 , Infecções por Coronavirus/transmissão , Desenho de Equipamento , Feminino , Filtração/métodos , Pessoal de Saúde , Humanos , Masculino , Pneumonia Viral/transmissão , Infecções Respiratórias/transmissão , SARS-CoV-2
14.
Anesth Analg ; 131(2): 411-448, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32467512

RESUMO

This consensus statement presents a comprehensive and evidence-based set of guidelines for the care of postoperative nausea and vomiting (PONV) in both adult and pediatric populations. The guidelines are established by an international panel of experts under the auspices of the American Society of Enhanced Recovery and Society for Ambulatory Anesthesia based on a comprehensive search and review of literature up to September 2019. The guidelines provide recommendation on identifying high-risk patients, managing baseline PONV risks, choices for prophylaxis, and rescue treatment of PONV as well as recommendations for the institutional implementation of a PONV protocol. In addition, the current guidelines focus on the evidence for newer drugs (eg, second-generation 5-hydroxytryptamine 3 [5-HT3] receptor antagonists, neurokinin 1 (NK1) receptor antagonists, and dopamine antagonists), discussion regarding the use of general multimodal PONV prophylaxis, and PONV management as part of enhanced recovery pathways. This set of guidelines have been endorsed by 23 professional societies and organizations from different disciplines (Appendix 1).Guidelines currently available include the 3 iterations of the consensus guideline we previously published, which was last updated 6 years ago; a guideline published by American Society of Health System Pharmacists in 1999; a brief discussion on PONV management as part of a comprehensive postoperative care guidelines; focused guidelines published by the Society of Obstetricians and Gynecologists of Canada, the Association of Paediatric Anaesthetists of Great Britain & Ireland and the Association of Perianesthesia Nursing; and several guidelines published in other languages.The current guideline was developed to provide perioperative practitioners with a comprehensive and up-to-date, evidence-based guidance on the risk stratification, prevention, and treatment of PONV in both adults and children. The guideline also provides guidance on the management of PONV within enhanced recovery pathways.The previous consensus guideline was published 6 years ago with a literature search updated to October 2011. Several guidelines, which have been published since, are either limited to a specific populations or do not address all aspects of PONV management. The current guideline was developed based on a systematic review of the literature published up through September 2019. This includes recent studies of newer pharmacological agents such as the second-generation 5-hydroxytryptamine 3 (5-HT3) receptor antagonists, a dopamine antagonist, neurokinin 1 (NK1) receptor antagonists as well as several novel combination therapies. In addition, it also contains an evidence-based discussion on the management of PONV in enhanced recovery pathways. We have also discussed the implementation of a general multimodal PONV prophylaxis in all at-risk surgical patients based on the consensus of the expert panel.


Assuntos
Consenso , Gerenciamento Clínico , Náusea e Vômito Pós-Operatórios/terapia , Guias de Prática Clínica como Assunto/normas , Acetaminofen/administração & dosagem , Administração Intravenosa , Analgésicos não Narcóticos/administração & dosagem , Antieméticos/administração & dosagem , Humanos , Náusea e Vômito Pós-Operatórios/diagnóstico
15.
Paediatr Anaesth ; 30(2): 124-136, 2020 02.
Artigo em Inglês | MEDLINE | ID: mdl-31841242

RESUMO

BACKGROUND: X-linked adrenoleukodystrophy is a progressive demyelinating disease that primarily affects males with an incidence of 1:20 000-30 000. The disease has a wide spectrum of phenotypic expression and may include adrenal insufficiency, cerebral X-linked adrenoleukodystrophy and adrenomyeloneuropathy. The condition has implications for the administration of anesthesia and reports of anesthetic management in those patients are limited at this point. AIM: To review the perioperative care, complications and outcomes of patients diagnosed with X-linked adrenoleukodystrophy at the University of Minnesota Masonic Children's Hospital. METHOD: After obtaining IRB approval, we performed a retrospective chart review of pediatric patients diagnosed with X-linked adrenoleukodystrophy who underwent either surgery or diagnostic/therapeutic procedures that included anesthesia services between January 2014 and December 2016. Data included demographics, American Society of Anesthesiologists classification, preoperative diagnosis, history of hematopoietic stem cell transplant, anesthetic approaches, airway management, medications used, intra- and postoperative complications, and patient disposition. RESULTS: We identified 38 patients who had a total of 166 anesthetic encounters. The majority of patients underwent procedures in the sedation unit (75.9%) and received a total intravenous anesthetic with spontaneous ventilation via a natural airway (86.1%). Preoperative adrenal insufficiency was documented in 87.3% of the encounters. Stress-dose steroids were administered in 70.5% of the performed anesthetics. A variety of anesthetic agents were successfully used including sevoflurane, isoflurane, propofol, midazolam, ketamine, and dexmedetomidine. There were few perioperative complications noted (6.6%) and the majority were of low severity. No anesthesia-related mortality was observed. CONCLUSIONS: With the availability of skilled pediatric anesthesia care, children with X-linked adrenoleukodystrophy can undergo procedures under anesthesia in sedation units and regular operating rooms with low overall anesthesia risk.


Assuntos
Adrenoleucodistrofia/cirurgia , Anestesia/métodos , Assistência Perioperatória/métodos , Adolescente , Anestésicos Inalatórios , Anestésicos Intravenosos , Criança , Pré-Escolar , Feminino , Humanos , Hipnóticos e Sedativos , Lactente , Masculino , Estudos Retrospectivos
16.
J Anaesthesiol Clin Pharmacol ; 36(4): 552-555, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33840940

RESUMO

Malignant hyperthermia susceptibility (MHS) and the associated condition malignant hyperthermia (MH) are rare but well-known disorders in the field of anesthesiology. MHS is usually determined by a history of a family member developing a positive episode during general anesthesia and then confirmed by an invasive caffeine halothane contracture test (CHCT). More recently, within the context of MH as a pharmacogenetic disorder, the question of whether or not MHS can be principally genetically determined is of high importance as knowledge of detailed pathogenesis may prevent against its largely invariable lethality if untreated. Thus, in this brief report, genetic terms, as well as updates in the genetics of MHS, will be reviewed in order to better understand both the condition and the current research.

19.
Anesth Analg ; 129(2): 347-349, 2019 08.
Artigo em Inglês | MEDLINE | ID: mdl-31166228

RESUMO

This document represents a joint effort of the Society for Ambulatory Anesthesia (SAMBA) and the Ambulatory Surgical Care Committee of the American Society of Anesthesiologists (ASA) concerning the safe anesthetic care of adult malignant hyperthermia (MH)-susceptible patients in a free-standing ambulatory surgery center (ASC). Adult MH-susceptible patients can safely undergo a procedure in a free-standing ASC assuming that proper precautions for preventing, identifying, and managing MH are taken. The administration of preoperative prophylaxis with dantrolene is not indicated in MH-susceptible patients scheduled for elective surgery. There is no evidence to recommend an extended stay in the ASC, and the patient may be discharged when the usual discharge criteria for outpatient surgery are met. Survival from an MH crisis in an ASC setting requires early recognition, prompt treatment, and timely transfer to a center with critical care capabilities.


Assuntos
Procedimentos Cirúrgicos Ambulatórios/normas , Anestesia/normas , Hospitalização , Hipertermia Maligna/terapia , Centros Cirúrgicos/normas , Procedimentos Cirúrgicos Ambulatórios/efeitos adversos , Anestesia/efeitos adversos , Dantroleno/administração & dosagem , Diagnóstico Precoce , Humanos , Hipertermia Maligna/diagnóstico , Hipertermia Maligna/etiologia , Relaxantes Musculares Centrais/administração & dosagem , Transferência de Pacientes/normas , Medição de Risco , Fatores de Risco , Resultado do Tratamento
20.
Orphanet J Rare Dis ; 14(1): 137, 2019 06 13.
Artigo em Inglês | MEDLINE | ID: mdl-31196221

RESUMO

INTRODUCTION: Mucopolysaccharidosis (MPS) IVA or Morquio A syndrome is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of the N-acetylgalactosamine-6-sulfatase (GALNS) enzyme, which impairs lysosomal degradation of keratan sulphate and chondroitin-6-sulphate. The multiple clinical manifestations of MPS IVA present numerous challenges for management and necessitate the need for individualised treatment. Although treatment guidelines are available, the methodology used to develop this guidance has come under increased scrutiny. This programme was conducted to provide evidence-based, expert-agreed recommendations to optimise management of MPS IVA. METHODS: Twenty six international healthcare professionals across multiple disciplines, with expertise in managing MPS IVA, and three patient advocates formed the Steering Committee (SC) and contributed to the development of this guidance. Representatives from six Patient Advocacy Groups (PAGs) were interviewed to gain insights on patient perspectives. A modified-Delphi methodology was used to demonstrate consensus among a wider group of healthcare professionals with experience managing patients with MPS IVA and the manuscript was evaluated against the validated Appraisal of Guidelines for Research and Evaluation (AGREE II) instrument by three independent reviewers. RESULTS: A total of 87 guidance statements were developed covering five domains: (1) general management principles; (2) recommended routine monitoring and assessments; (3) disease-modifying interventions (enzyme replacement therapy [ERT] and haematopoietic stem cell transplantation [HSCT]); (4) interventions to support respiratory and sleep disorders; (5) anaesthetics and surgical interventions (including spinal, limb, ophthalmic, cardio-thoracic and ear-nose-throat [ENT] surgeries). Consensus was reached on all statements after two rounds of voting. The overall guideline AGREE II assessment score obtained for the development of the guidance was 5.3/7 (where 1 represents the lowest quality and 7 represents the highest quality of guidance). CONCLUSION: This manuscript provides evidence- and consensus-based recommendations for the management of patients with MPS IVA and is for use by healthcare professionals that manage the holistic care of patients with the intention to improve clinical- and patient-reported outcomes and enhance patient quality of life. It is recognised that the guidance provided represents a point in time and further research is required to address current knowledge and evidence gaps.


Assuntos
Condroitina Sulfatases/metabolismo , Mucopolissacaridose IV/metabolismo , Condroitina Sulfatases/genética , Terapia de Reposição de Enzimas/métodos , Feminino , Humanos , Hipercapnia/genética , Hipercapnia/metabolismo , Masculino
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