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Assessment, endoscopy, and treatment in patients with acute severe ulcerative colitis during the COVID-19 pandemic (PROTECT-ASUC): a multicentre, observational, case-control study (2021)
Journal Article
Sebastian, S., Walker, G. J., Kennedy, N. A., Conley, T. E., Patel, K. V., Subramanian, S., …Zafar, M. (2021). Assessment, endoscopy, and treatment in patients with acute severe ulcerative colitis during the COVID-19 pandemic (PROTECT-ASUC): a multicentre, observational, case-control study. Lancet Gastroenterology and Hepatology, 6(4), 271-281. https://doi.org/10.1016/s2468-1253%2821%2900016-9

Background There is a paucity of evidence to support safe and effective management of patients with acute severe ulcerative colitis during the COVID-19 pandemic. We sought to identify alterations to established conventional evidence-based management... Read More about Assessment, endoscopy, and treatment in patients with acute severe ulcerative colitis during the COVID-19 pandemic (PROTECT-ASUC): a multicentre, observational, case-control study.

MATRix–RICE therapy and autologous haematopoietic stem-cell transplantation in diffuse large B-cell lymphoma with secondary CNS involvement (MARIETTA): an international, single-arm, phase 2 trial (2021)
Journal Article
Ferreri, A. J. M., Doorduijn, J. K., Re, A., Cabras, M. G., Smith, J., Ilariucci, F., …International Extranodal Lymphoma Study Group (IELSG), I. E. L. S. G. (. (2021). MATRix–RICE therapy and autologous haematopoietic stem-cell transplantation in diffuse large B-cell lymphoma with secondary CNS involvement (MARIETTA): an international, single-arm, phase 2 trial. Lancet Haematology, 8(2), e110-e121. https://doi.org/10.1016/S2352-3026%2820%2930366-5

Background: Secondary CNS lymphoma is a rare but potentially lethal event in patients with diffuse large B-cell lymphoma. We aimed to assess the activity and safety of an intensive, CNS-directed chemoimmunotherapy consolidated by autologous haematopo... Read More about MATRix–RICE therapy and autologous haematopoietic stem-cell transplantation in diffuse large B-cell lymphoma with secondary CNS involvement (MARIETTA): an international, single-arm, phase 2 trial.

A systematic review and meta-analysis of Anakinra, Sarilumab, Siltuximab and Tocilizumab for Covid-19 (2021)
Preprint / Working Paper
Khan, F., Fabbri, L., Stewart, I., Robinson A., K., Smyth, A. R., & Jenkins, G. A systematic review and meta-analysis of Anakinra, Sarilumab, Siltuximab and Tocilizumab for Covid-19

Background There is accumulating evidence for an overly activated immune response in severe Covid-19, with several studies exploring the therapeutic role of immunomodulation. Through systematic review and meta-analysis, we assess the effectiveness of... Read More about A systematic review and meta-analysis of Anakinra, Sarilumab, Siltuximab and Tocilizumab for Covid-19.

A phase 1/2 study of the combination of acalabrutinib and vistusertib in patients with relapsed/refractory B-cell malignancies (2021)
Journal Article
Collins, G. P., Clevenger, T. N., Burke, K. A., Yang, B., MacDonald, A., Cunningham, D., …Flinn, I. (2021). A phase 1/2 study of the combination of acalabrutinib and vistusertib in patients with relapsed/refractory B-cell malignancies. Leukemia & Lymphoma, 62(11), 2625-2636. https://doi.org/10.1080/10428194.2021.1938027

In a phase 1b study of acalabrutinib (a covalent Bruton tyrosine kinase (BTK) inhibitor) in combination with vistusertib (a dual mTORC1/2 inhibitor) in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), multiple ascending doses... Read More about A phase 1/2 study of the combination of acalabrutinib and vistusertib in patients with relapsed/refractory B-cell malignancies.

ERS international congress 2020: Highlights from the paediatric assembly (2021)
Journal Article
Ardura-Garcia, C., Cuevas-Ocaña, S., Freitag, N., Kampouras, A., King, J. A., Kouis, P., …Pijnenburg, M. W. (2021). ERS international congress 2020: Highlights from the paediatric assembly. ERJ Open Research, 7(1), Article 00893-2020. https://doi.org/10.1183/23120541.00893-2020

In this review, the Paediatric Assembly of the European Respiratory Society (ERS) presents a summary of the highlights and most relevant findings in the field of paediatric respiratory medicine presented at the virtual ERS International Congress 2020... Read More about ERS international congress 2020: Highlights from the paediatric assembly.

RAD50 deficiency is a predictor of platinum sensitivity in sporadic epithelial ovarian cancers (2020)
Journal Article
Alblihy, A., Alabdullah, M. L., Toss, M. S., Algethami, M., Mongan, N. P., Rakha, E. A., & Madhusudan, S. (2020). RAD50 deficiency is a predictor of platinum sensitivity in sporadic epithelial ovarian cancers. Molecular Biomedicine, 1(1), Article 19. https://doi.org/10.1186/s43556-020-00023-y

Intrinsic or acquired resistance seriously limits the use of platinating agents in advanced epithelial ovarian cancers. Increased DNA repair capacity is a key route to platinum resistance. RAD50 is a critical component of the MRN complex, a ‘first re... Read More about RAD50 deficiency is a predictor of platinum sensitivity in sporadic epithelial ovarian cancers.

Preferential transcription of the mutated allele in NPM1 mutated acute myeloid leukaemia (2020)
Journal Article
Bailey, G. D., Doolan, L., Baskar, A., Smith, L. C., & Seedhouse, C. H. (2020). Preferential transcription of the mutated allele in NPM1 mutated acute myeloid leukaemia. Scientific Reports, 10, Article 17695. https://doi.org/10.1038/s41598-020-73782-x

Nucleophosmin is commonly both over-expressed and mutated in acute myeloid leukemia (AML). NPM1 mutations are always heterozygous. In addition, NPM1 has a number of different splice variants with the major variant encoded by exons 1–9 and 11–12 (NPM1... Read More about Preferential transcription of the mutated allele in NPM1 mutated acute myeloid leukaemia.

The era of CFTR modulators: improvements made and remaining challenges (2020)
Journal Article
Cuevas-Ocaña, S., Laselva, O., Avolio, J., & Nenna, R. (2020). The era of CFTR modulators: improvements made and remaining challenges. Breathe, 16(2), Article 200016. https://doi.org/10.1183/20734735.0016-2020

This last decade has created historical moments for CF, primarily driven by the development of CFTR modulators. First for patients with gating mutations who benefited from Kalydeco, then for those patients with one F508del copy who could benefit from... Read More about The era of CFTR modulators: improvements made and remaining challenges.

Inter- and Intra-individual Variation, and Limited Prognostic Utility, of Serum Alkaline Phosphatase in a Trial of Patients With Primary Sclerosing Cholangitis (2020)
Journal Article
Trivedi, P. J., Muir, A. J., Levy, C., Bowlus, C. L., Manns, M. P., Lu, X., …Hirschfield, G. M. (2021). Inter- and Intra-individual Variation, and Limited Prognostic Utility, of Serum Alkaline Phosphatase in a Trial of Patients With Primary Sclerosing Cholangitis. Clinical Gastroenterology and Hepatology, 19(6), 1248-1257. https://doi.org/10.1016/j.cgh.2020.07.032

Background & Aims Serum alkaline phosphatase (ALP) and the enhanced liver fibrosis (ELF) score are used as endpoints in trials of patients with primary sclerosing cholangitis (PSC). We aimed to quantify inter- and intra-individual variation in level... Read More about Inter- and Intra-individual Variation, and Limited Prognostic Utility, of Serum Alkaline Phosphatase in a Trial of Patients With Primary Sclerosing Cholangitis.

Association of Sperm-Associated Antigen 5 and Treatment Response in Patients With Estrogen Receptor–Positive Breast Cancer (2020)
Journal Article
Abdel-Fatah, T. M., Ball, G. R., Thangavelu, P. U., Reid, L. E., Reed, A. E. M., Saunus, J. M., …Chan, S. Y. (2020). Association of Sperm-Associated Antigen 5 and Treatment Response in Patients With Estrogen Receptor–Positive Breast Cancer. JAMA Network Open, 3(7), Article e209486. https://doi.org/10.1001/jamanetworkopen.2020.9486

Importance There is no proven test that can guide the optimal treatment, either endocrine therapy or chemotherapy, for estrogen receptor–positive breast cancer. Objective To investigate the associations of sperm-associated antigen 5 (SPAG5) tran... Read More about Association of Sperm-Associated Antigen 5 and Treatment Response in Patients With Estrogen Receptor–Positive Breast Cancer.

Epithelial cell dysfunction, a major driver of asthma development (2020)
Journal Article
Heijink, I. H., Kuchibhotla, V. N. S., Roffel, M. P., Maes, T., Knight, D. A., Sayers, I., & Nawijn, M. C. (2020). Epithelial cell dysfunction, a major driver of asthma development. Allergy, 75(8), 1902-1917. https://doi.org/10.1111/all.14421

Airway epithelial barrier dysfunction is frequently observed in asthma and may have important implications. The physical barrier function of the airway epithelium is tightly interwoven with its immunomodulatory actions, while abnormal epithelial repa... Read More about Epithelial cell dysfunction, a major driver of asthma development.

Discovery of highly selective inhibitors of calmodulin-dependent kinases that restore insulin sensitivity in the diet-induced obese in vivo mouse model. (2020)
Journal Article
Fromont, C., Atzori, A., Kaur, D., Hashmi, L., Greco, G., Cabanillas, A., …Butterworth, S. (2020). Discovery of highly selective inhibitors of calmodulin-dependent kinases that restore insulin sensitivity in the diet-induced obese in vivo mouse model. Journal of Medicinal Chemistry, 63(13), 6784–6801. https://doi.org/10.1021/acs.jmedchem.9b01803

ERS international congress, madrid, 2019: Highlights from the airway diseases, asthma and COPD assembly (2020)
Journal Article
Lahousse, L., Bahmer, T., Cuevas-Ocaña, S., Flajolet, P., Mathioudakis, A. G., McDonnell, M., …Bonvini, S. J. (2020). ERS international congress, madrid, 2019: Highlights from the airway diseases, asthma and COPD assembly. ERJ Open Research, 6(1), https://doi.org/10.1183/23120541.00341-2019

The European Respiratory Society (ERS) International Congress 2019 in Madrid, Spain, was a platform for scientific discussion of the highest quality scientific research, cutting-edge techniques and innovative new therapies within the respiratory fiel... Read More about ERS international congress, madrid, 2019: Highlights from the airway diseases, asthma and COPD assembly.

FibroScan-AST (FAST) score for the non-invasive identification of patients with non-alcoholic steatohepatitis with significant activity and fibrosis: a prospective derivation and global validation study (2020)
Journal Article
Newsome, P. N., Sasso, M., Deeks, J. J., Paredes, A., Boursier, J., Chan, W.-K., …Harrison, S. A. (2020). FibroScan-AST (FAST) score for the non-invasive identification of patients with non-alcoholic steatohepatitis with significant activity and fibrosis: a prospective derivation and global validation study. The Lancet Gastroenterology & Hepatology, 5(4), 362-373. https://doi.org/10.1016/S2468-1253%2819%2930383-8

Background The burden of non-alcoholic fatty liver disease (NAFLD) is increasing globally, and a major priority is to identify patients with non-alcoholic steatohepatitis (NASH) who are at greater risk of progression to cirrhosis, and who will be ca... Read More about FibroScan-AST (FAST) score for the non-invasive identification of patients with non-alcoholic steatohepatitis with significant activity and fibrosis: a prospective derivation and global validation study.

How can gene-editing of human pluripotent stem cells help cystic fibrosis? (2019)
Presentation / Conference Contribution
Cuevas Ocana, S., Wong, A., Aushev, M., Ye Yang, J., Perkins, N., Bear, C., …Gray, M. (2019). How can gene-editing of human pluripotent stem cells help cystic fibrosis?. European Respiratory Journal, 54(suppl 63), Article OA2125. https://doi.org/10.1183/13993003.congress-2019.oa2125

Cystic fibrosis (CF) is a monogenic recessive disorder, affecting 70,000 people worldwide. CF is due to mutations in the CFTR gene, resulting in a defective protein that leads to symptoms in numerous organs, especially lungs. Despite recent advances,... Read More about How can gene-editing of human pluripotent stem cells help cystic fibrosis?.

Anticholinergic Drug Exposure and the Risk of Dementia: A Nested Case-Control Study (2019)
Journal Article
Coupland, C. A., Hill, T., Dening, T., Morriss, R., Moore, M., & Hippisley-Cox, J. (2019). Anticholinergic Drug Exposure and the Risk of Dementia: A Nested Case-Control Study. JAMA Internal Medicine, 179(8), 1084-1093. https://doi.org/10.1001/jamainternmed.2019.0677

IMPORTANCE Anticholinergic medicines have short-term cognitive adverse effects, but it is uncertain whether long-term use of these drugs is associated with an increased risk of dementia. OBJECTIVE To assess associations between anticholinergic dr... Read More about Anticholinergic Drug Exposure and the Risk of Dementia: A Nested Case-Control Study.

Drug Development for Nonalcoholic Fatty Liver Disease: Landscape and Challenges (2019)
Journal Article
Thiagarajan, P., & Aithal, G. P. (2019). Drug Development for Nonalcoholic Fatty Liver Disease: Landscape and Challenges. Journal of Clinical and Experimental Hepatology, 9(4), 515-521. https://doi.org/10.1016/j.jceh.2019.03.002

Nonalcoholic fatty liver disease (NAFLD) is now the leading cause of chronic liver disease in industrialized economies. With no licensed treatment currently available, together with a growing prevalence that parallels global increases in obesity and... Read More about Drug Development for Nonalcoholic Fatty Liver Disease: Landscape and Challenges.

Refractoriness to rituximab-based therapy and elevated serum B2-microglobulin predict for inferior survival in marginal zone lymphoma (2019)
Journal Article
Sorigue, M., Bishton, M., Domingo-Domenech, E., McMillan, A., Prusila, R., García, O., …Sancho, J. (2019). Refractoriness to rituximab-based therapy and elevated serum B2-microglobulin predict for inferior survival in marginal zone lymphoma. Leukemia & Lymphoma, 60(10), 2524-2531. https://doi.org/10.1080/10428194.2019.1594212

Short responses to immunochemotherapy predict for an inferior OS in follicular lymphoma. We set out to determine whether this is also the case in marginal zone lymphoma. A group of 139 marginal zone lymphoma (MZL) patients treated with front-line imm... Read More about Refractoriness to rituximab-based therapy and elevated serum B2-microglobulin predict for inferior survival in marginal zone lymphoma.