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61. Dinutuximab beta for treating neuroblastoma

and debilitating, with severe and long-lasting side effects (including hearing loss, organ dysfunction, sterility, lack of growth, early onset of puberty, permanent disability, and secondary malignancies). The clinical and patient experts stated that a child's death has a significant effect on family members. The committee noted that treatment can involve many hospital visits and stays causing disruption to school, work and family life. It understood that parents and carers also have anxiety, emotional (...) acknowledged that there were uncaptured Dinutuximab beta for treating neuroblastoma (TA538) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 20 of 25health-related benefits. These included reduced quality of life because of the effect of stress and depression caused by the disease on young patients and their families, as well as the devastating effects of bereavement on families. It discussed the need to balance

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

62. Burosumab for treating X-linked hypophosphataemia in children and young people

Evaluation committee members 29 NICE project team 29 Burosumab for treating X-linked hypophosphataemia in children and young people (HST8) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 301 1 Recommendations Recommendations 1.1 Burosumab is recommended, within its marketing authorisation, for treating X-linked hypophosphataemia (XLH) with radiographic evidence of bone disease in children aged 1 year and over (...) to errors in phosphate sensing and increased levels of fibroblast growth factor 23 (FGF23). Excess FGF23 causes impaired phosphate conservation and excessive phosphate excretion. It also supresses vitamin D production, which causes reduced calcium and phosphate absorption. 2.2 Because XLH is a genetic condition, it often affects several members of a family. Skeletal abnormalities such as bowed or bent legs, below average height and irregular growth of the skull are early signs of XLH. Children may also

2018 National Institute for Health and Clinical Excellence - Highly specialised technology

63. Avelumab for treating metastatic Merkel cell carcinoma

6 Appraisal committee members and NICE project team 23 Appraisal committee members 23 NICE project team 23 Avelumab for treating metastatic Merkel cell carcinoma (TA517) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 241 1 Recommendations Recommendations 1.1 Avelumab is recommended as an option for treating metastatic Merkel cell carcinoma in adults, only if they have had 1 or more lines of chemotherapy (...) Avelumab for treating metastatic Merkel cell carcinoma A Av velumab for treating metastatic Merk elumab for treating metastatic Merkel el cell carcinoma cell carcinoma T echnology appraisal guidance Published: 11 April 2018 nice.org.uk/guidance/ta517 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE, arrived

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

64. Eribulin for treating locally advanced or metastatic breast cancer after 1 chemotherapy regimen

://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 2 of 17Contents Contents 1 Recommendations 4 2 The technology 6 3 Committee discussion 7 Symptoms and management of advanced breast cancer 7 Clinical trial evidence 8 The economic model 11 Clinical parameters 11 Costs 14 Cost-effectiveness estimates 14 End of life 15 Other factors 15 4 Appraisal committee members and NICE project team 16 Appraisal committee members 16 NICE project team 16 Eribulin for treating locally advanced or metastatic breast (...) and convenient administration method. However, the committee concluded that it could not identify any specific health-related benefit that had not already been captured in the QAL Y calculation. Eribulin for treating locally advanced or metastatic breast cancer after 1 chemotherapy regimen (TA515) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 15 of 174 4 Appr Appraisal committee members and NICE project team aisal

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

65. Strimvelis for treating adenosine deaminase deficiency?severe combined immunodeficiency

cell donor is available. Wh Why the committee made this recommendation y the committee made this recommendation ADA–SCID is a rare and serious condition that is fatal if untreated, and which severely affects the quality of life of people with the condition and their families. Treatment for ADA–SCID includes haematopoietic stem cell transplants (HSCTs). Clinical trial evidence shows that Strimvelis is effective in treating ADA–SCID. Compared with HSCTs, results suggest the main benefits (...) condition that also severely affects the lives of families and carers. Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 8 of 31Diagnosis and management Diagnosis and management 4.3 The committee heard from clinical experts that, when ADA–SCID is suspected, people will be referred to 1 of the 2 expert centres in England, where

2018 National Institute for Health and Clinical Excellence - Highly specialised technology

66. Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma

for data collection 19 6 Appraisal committee members and NICE project team 20 Appraisal committee members 20 NICE project team 20 Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma (TA505) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 211 1 Recommendations Recommendations 1.1 Ixazomib, with lenalidomide and dexamethasone, is recommended for use within the Cancer (...) -1 clinical trial. Data on overall survival and time-on- treatment will be collected through other sources including the Systemic Anti- Cancer Therapy (SACT) dataset. Ixazomib with lenalidomide and dexamethasone for treating relapsed or refractory multiple myeloma (TA505) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 19 of 216 6 Appr Appraisal committee members and NICE project team aisal committee members

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

67. Pirfenidone for treating idiopathic pulmonary fibrosis

discussion 7 Review objectives 7 Current practice 7 Comparators 8 Clinical effectiveness 8 Cost effectiveness 11 Cost-effectiveness results and conclusions 16 Potential equality issues 18 The Pharmaceutical Price Regulation Scheme 19 Summary of appraisal committee's key conclusions 19 5 Implementation 24 6 Appraisal committee members, guideline representatives and NICE project team 25 Appraisal committee members 25 NICE project team 25 Pirfenidone for treating idiopathic pulmonary fibrosis (TA504) © NICE (...) Pirfenidone for treating idiopathic pulmonary fibrosis Pirfenidone for treating idiopathic Pirfenidone for treating idiopathic pulmonary fibrosis pulmonary fibrosis T echnology appraisal guidance Published: 6 February 2018 nice.org.uk/guidance/ta504 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE, arrived

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

68. Ibrutinib for treating relapsed or refractory mantle cell lymphoma

3 Evidence 6 4 Committee discussion 7 Clinical effectiveness 7 Cost effectiveness 11 End-of-life considerations 13 Potential equality issues 14 Pharmaceutical Price Regulation Scheme (PPRS) 2014 14 Summary of appraisal committee's key conclusions 14 5 Implementation 20 6 Appraisal committee members and NICE project team 21 Appraisal committee members 21 NICE project team 21 Ibrutinib for treating relapsed or refractory mantle cell lymphoma (TA502) © NICE 2018. All rights reserved. Subject (...) Ibrutinib for treating relapsed or refractory mantle cell lymphoma Ibrutinib for treating relapsed or Ibrutinib for treating relapsed or refr refractory mantle cell lymphoma actory mantle cell lymphoma T echnology appraisal guidance Published: 31 January 2018 nice.org.uk/guidance/ta502 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

69. Dupilumab for treating moderate to severe atopic dermatitis

and NICE project team 20 Appraisal committee members 20 NICE project team 20 Dupilumab for treating moderate to severe atopic dermatitis (TA534) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 3 of 211 1 Recommendations Recommendations 1.1 Dupilumab is recommended as an option for treating moderate to severe atopic dermatitis in adults, only if: the disease has not responded to at least 1 other systemic therapy (...) Dupilumab for treating moderate to severe atopic dermatitis Dupilumab for treating moder Dupilumab for treating moderate to ate to se sev vere atopic dermatitis ere atopic dermatitis T echnology appraisal guidance Published: 1 August 2018 nice.org.uk/guidance/ta534 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view of NICE

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

70. Ocrelizumab for treating relapsing?remitting multiple sclerosis

responsible for their care thinks that ocrelizumab is the right treatment, it should be available for use, in line with NICE's recommendations. Ocrelizumab for treating relapsing–remitting multiple sclerosis (TA533) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 20 of 225 5 Appr Appraisal committee members and NICE project team aisal committee members and NICE project team Appraisal committee members The 4 technology (...) Ocrelizumab for treating relapsing?remitting multiple sclerosis Ocrelizumab for treating Ocrelizumab for treating relapsing–remitting multiple sclerosis relapsing–remitting multiple sclerosis T echnology appraisal guidance Published: 25 July 2018 nice.org.uk/guidance/ta533 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility The recommendations in this guidance represent the view

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

71. Lenvatinib and sorafenib for treating differentiated thyroid cancer after radioactive iodine

Resource use 16 Treatment after disease progression 17 Revised base case 17 Cost-effectiveness results 18 Uncaptured benefits 18 End of life 19 Conclusions 20 Other factors 22 4 Implementation 23 5 Appraisal committee members and NICE project team 24 Appraisal committee members 24 NICE project team 24 Lenvatinib and sorafenib for treating differentiated thyroid cancer after radioactive iodine (TA535) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms (...) Lenvatinib and sorafenib for treating differentiated thyroid cancer after radioactive iodine L Len envatinib and sor vatinib and sorafenib for treating afenib for treating differentiated th differentiated thyroid cancer after yroid cancer after r radioactiv adioactive iodine e iodine T echnology appraisal guidance Published: 8 August 2018 nice.org.uk/guidance/ta535 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

72. Pertuzumab with trastuzumab and docetaxel for treating HER2-positive breast cancer

-of-rights). Page 2 of 18Contents Contents 1 Recommendations 4 2 The technology 5 3 Evidence 6 4 Committee discussion 7 Clinical effectiveness 9 Cost effectiveness 10 Innovation 12 End-of-life considerations 13 Conclusions 14 5 Implementation 16 6 Appraisal committee members and NICE project team 17 Appraisal committee members 17 NICE project team 17 Pertuzumab with trastuzumab and docetaxel for treating HER2-positive breast cancer (TA509) © NICE 2018. All rights reserved. Subject to Notice of rights (...) enquiries from NHS organisations about the commercial access agreement should be directed to global.pas@roche.com. Pertuzumab with trastuzumab and docetaxel for treating HER2-positive breast cancer (TA509) © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and- conditions#notice-of-rights). Page 16 of 186 6 Appr Appraisal committee members and NICE project team aisal committee members and NICE project team Appraisal committee members The technology appraisal

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

73. Daratumumab monotherapy for treating relapsed and refractory multiple myeloma

of 31Contents Contents 1 Recommendations 4 2 Information about daratumumab 5 3 Evidence 6 4 Committee discussion 7 Nature of the condition 7 Clinical pathway 7 Clinical effectiveness 8 Cost effectiveness 16 Innovation 20 End-of-life considerations 21 Cancer Drugs Fund considerations 21 Summary of appraisal committee's key conclusions 24 5 Implementation 29 6 Appraisal committee members and NICE project team 30 Appraisal committee members 30 NICE project team 30 Daratumumab monotherapy for treating relapsed (...) Daratumumab monotherapy for treating relapsed and refractory multiple myeloma Dar Daratumumab monother atumumab monotherap apy for treating y for treating relapsed and refr relapsed and refractory multiple actory multiple m my yeloma eloma T echnology appraisal guidance Published: 14 March 2018 nice.org.uk/guidance/ta510 © NICE 2018. All rights reserved. Subject to Notice of rights (https://www.nice.org.uk/terms-and-conditions#notice-of- rights).Y Y our responsibility our responsibility

2018 National Institute for Health and Clinical Excellence - Technology Appraisals

74. WITHDRAWN: Interventions for treating obesity in children. Full Text available with Trip Pro

checked. No language restrictions were applied.We selected randomised controlled trials (RCTs) of lifestyle (i.e. dietary, physical activity and/or behavioural therapy), drug and surgical interventions for treating obesity in children (mean age under 18 years) with or without the support of family members, with a minimum of six months follow up (three months for actual drug therapy). Interventions that specifically dealt with the treatment of eating disorders or type 2 diabetes, or included (...) WITHDRAWN: Interventions for treating obesity in children. Child and adolescent obesity is increasingly prevalent, and can be associated with significant short- and long-term health consequences.To assess the efficacy of lifestyle, drug and surgical interventions for treating obesity in childhood.We searched CENTRAL on The Cochrane Library Issue 2 2008, MEDLINE, EMBASE, CINAHL, PsycINFO, ISI Web of Science, DARE and NHS EED. Searches were undertaken from 1985 to May 2008. References were

2019 Cochrane

75. Guest Post: Family doctors can easily treat hepatitis B "in-house"

Guest Post: Family doctors can easily treat hepatitis B "in-house" The AFP Community Blog: Guest Post: Family doctors can easily treat hepatitis B "in-house" | Monday, March 11, 2019 - Richard Andrews, MD, MPH In 2008, I replaced another family physician as the sole physician at , a federally qualified health center in southwest Houston. The clinic was founded by members of the local Asian community to ensure that Asians would have a place to receive linguistically and culturally-appropriate (...) , they were reflexively referred to hepatologists, but due to access and cost barriers, the community asked us to start treating hepatitis B right at HOPE. I was an experienced, board-certified family physician, but I had never done any hepatitis B management. I sought training with local hepatologists, mostly through lectures and question-and-answer sessions, and gradually became comfortable with the vocabulary, epidemiology, concepts, and pharmacology of . Because of the HOPE Clinic's origins, most

2019 The AFP Community Blog

76. Management of patients with increased risk for familial pancreatic cancer

with pancreatic cancer can help to identify family members who may benefit from surveillance. The average lifetime risk of developing pancreatic cancer (~1 in 64 in the USA) is too low for population- based screening. 20 21 Germline mutation carriers Pancreatic surveillance is recommended for carriers of germline deleterious variants in cancer susceptibility gene, 22–26 : BRCA2, ATM, BRCA1, PALB2, CDKN2A, STK11, MLH1 and MSH2. Recommendations for age and family history vary by gene. Surveillance for CDKN2A (...) Management of patients with increased risk for familial pancreatic cancer 1 Goggins M, et al. Gut 2019;0:1–11. doi:10.1136/gutjnl-2019-319352 Guidelines Management of patients with increased risk for familial pancreatic cancer: updated recommendations from the International Cancer of the Pancreas Screening (CAPS) Consortium Michael Goggins , 1 Kasper Alexander Overbeek , 2 Randall Brand, 3 Sapna Syngal, 4 Marco Del Chiaro, 5 Detlef K Bartsch, 6 Claudio Bassi, 7 Alfredo Carrato, 8 James Farrell

2019 British Society of Gastroenterology

77. Understanding genetic tests for men who have a family history of breast and ovarian cancer

technology cannot find all of the variations that make the BRCA1 or BRCA2 genes faulty • your family history of cancer could be due to a combination of many factors (both genetic and environmental) If you receive an inconclusive result, other members of your family cannot be offered a predictive test.19 2) A faulty gene was found If you receive this result: • you have inherited a faulty cancer protection gene • you have an increased chance of developing another cancer • other blood relatives who have (...) ‘No’ to the test Your decision might be... A faulty BRCA1 or BRCA2 gene was found Inconclusive result or uncertain variant You might decide to have a genetic test at a later time Sign a consent form and then have a blood test Attend genetic counselling to receive test results You might decide to consider your testing options again in the future It is possible to offer a predictive test to other family members It is not possible to offer a predictive test to other family members ‘Yes’ to the test Defer

2019 European Society of Endocrinology

78. Perceptions of HIV transmission and pre-exposure prophylaxis among health care workers and community members in Rwanda. Full Text available with Trip Pro

workers felt that promotion of safe sex practices (74.5%), HIV testing and treating HIV infected patients (60.8%) would work better than PrEP to decrease new HIV infections. Barriers to PrEP implementation included perceived stigma, delayed access to prevention services at the health facilities while personal-level concerns included lack of family support, reluctance to take a medication daily and fear of being perceived as having HIV. This study showed that health care workers and community members (...) Perceptions of HIV transmission and pre-exposure prophylaxis among health care workers and community members in Rwanda. There are too many new HIV infections globally with 1.8 million persons infected in 2016 alone. Pre-exposure prophylaxis (PrEP) holds potential to decrease new infections and is synergistic with efforts currently in place to achieve an end to the AIDS epidemic in Sub-Saharan African, but uptake is limited. Given its novelty, assessing the beliefs and attitudes of healthcare

2018 PLoS ONE

79. Quality of life effects of androgen deprivation therapy in a prostate cancer cohort in New Zealand: can we minimize effects using a stratification based on the aldo-keto reductase family 1, member C3 rs12529 gene polymorphism? Full Text available with Trip Pro

Quality of life effects of androgen deprivation therapy in a prostate cancer cohort in New Zealand: can we minimize effects using a stratification based on the aldo-keto reductase family 1, member C3 rs12529 gene polymorphism? Androgen deprivation therapy (ADT) is an effective palliation treatment in men with advanced prostate cancer (PC). However, ADT has well documented side effects that could alter the patient's health-related quality of life (HRQoL). The current study aims to test whether (...) ) and the Depression Anxiety Stress Scales developed by the University of Melbourne, Australia. Genotyping of these men was carried out for the aldo-keto reductase family 1, member C3 (AKR1C3) rs12529 single nucleotide polymorphism (SNP). Analysis of HRQoL scores were carried out against ADT duration and in association with the AKR1C3 rs12529 SNP using the generalised linear model. P-values <0 · 05 were considered significant, and were further tested for restriction with Bonferroni correction.Increase in hormone

2016 BMC Urology

80. Influence of aging and growth hormone on different members of the NFkB family and IkB expression in the heart from a murine model of senescence-accelerated aging. (Abstract)

Influence of aging and growth hormone on different members of the NFkB family and IkB expression in the heart from a murine model of senescence-accelerated aging. Inflammation is related to several pathological processes. The aim of this study was to investigate the protein expression of the different subunits of the nuclear factor Kappa b (NFkBp65, p50, p105, p52, p100) and the protein expressions of IkB beta and alpha in the hearts from a murine model of accelerated aging (SAM model (...) ) by Western blot. In addition, the translocation of some isoforms of NFkB from cytosol to nuclei (NFkBp65, p50, p52) and ATP level content was studied. In addition we investigated the effect of the chronic administration of growth hormone (GH) on these age-related parameters. SAMP8 and SAMR1 mice of 2 and 10 months of age were used (n = 30). Animals were divided into five experimental groups: 2 old untreated (SAMP8/SAMR1), 2 young control (SAMP8/SAMR1) and one GH treated-old groups (SAMP8). Age-related

2016 Experimental Gerontology

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