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1. P196 Age at loss of ambulation in patients with DMD from the STRIDE registry and the CINRG natural history study: a matched cohort analysis

Author

Mercuri, E., primary, Muntoni, F., additional, Buccella, F., additional, Desguerre, I., additional, Kirschner, J., additional, Osorio, A Nascimento, additional, Tulinius, M., additional, de Resende, M., additional, Morgenroth, L., additional, Gordish-Dressman, H., additional, Johnson, S., additional, Werner, C., additional, Anbu, B., additional, Liu, E., additional, Rajbhandari, R., additional, Trifillis, P., additional, and McDonald, C., additional

Published
2023
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2. P198 Updated demographics and safety data from patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren in the STRIDE registry

Author

Muntoni, F., primary, Buccella, F., additional, Desguerre, I., additional, Kirschner, J., additional, Osorio, A Nascimento, additional, Tulinius, M., additional, de Resende, M., additional, Johnson, S., additional, Werner, C., additional, Anbu, B., additional, Liu, E., additional, Rajbhandari, R., additional, Trifillis, P., additional, and Mercuri, E., additional

Published
2023
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3. P197 Pulmonary function in patients with Duchenne muscular dystrophy from the STRIDE registry and CINRG natural history study: a matched cohort analysis

Author

Tulinius, M., primary, Buccella, F., additional, Desguerre, I., additional, Kirschner, J., additional, Mercuri, E., additional, Muntoni, F., additional, Osorio, A Nascimento, additional, de Resende, M., additional, Morgenroth, L., additional, Gordish-Dressman, H., additional, Johnson, S., additional, Werner, C., additional, Anbu, B., additional, Liu, E., additional, Rajbhandari, R., additional, Trifillis, P., additional, and McDonald, C., additional

Published
2023
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7. Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment.

Author

Roubertie, A., Leuzzi, V., Pearl, P. L., Ezgü, F., Lupo, P., Rajbhandari, R., Sierra, J. R., Grünert, J., and Giugliani, R.

Subjects
ACIDS, NEURAL development, EVERYDAY life
Abstract

This article provides an update on a two-part international observational registry called AADCAware, which focuses on individuals with aromatic L-amino acid decarboxylase deficiency (AADCd). AADCd is a rare neurodevelopmental disorder that causes severe impairment. Part A of the registry examines the natural history of AADCd in patients receiving standard care, while Part B assesses the long-term safety and efficacy of a specific treatment called eladocagene exuparvovec on motor function. The study found that the majority of subjects failed to achieve major motor milestones, indicating a significant need for daily life support. [Extracted from the article]

Published
2023
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24. CELL BIOLOGY AND SIGNALING

Author

Agarwal, M., primary, Nitta, R., additional, Dovat, S., additional, Li, G., additional, Arita, H., additional, Narita, Y., additional, Fukushima, S., additional, Tateishi, K., additional, Matsushita, Y., additional, Yoshida, A., additional, Miyakita, Y., additional, Ohno, M., additional, Collins, V. P., additional, Kawahara, N., additional, Shibui, S., additional, Ichimura, K., additional, Kahn, S. A., additional, Gholamin, S., additional, Junier, M.-P., additional, Chneiweiss, H., additional, Weissman, I., additional, Mitra, S., additional, Cheshier, S., additional, Avril, T., additional, Hamlat, A., additional, Le Reste, P.-J., additional, Mosser, J., additional, Quillien, V., additional, Carrato, C., additional, Munoz-Marmol, A., additional, Serrano, L., additional, Pijuan, L., additional, Hostalot, C., additional, Villa, S. l., additional, Ariza, A., additional, Etxaniz, O., additional, Balana, C., additional, Benveniste, E. T., additional, Zheng, Y., additional, McFarland, B., additional, Drygin, D., additional, Bellis, S., additional, Bredel, M., additional, Lotsch, D., additional, Engelmaier, C., additional, Allerstorfer, S., additional, Grusch, M., additional, Pichler, J., additional, Weis, S., additional, Hainfellner, J., additional, Marosi, C., additional, Spiegl-Kreinecker, S., additional, Berger, W., additional, Bronisz, A., additional, Nowicki, M. O., additional, Wang, Y., additional, Ansari, K., additional, Chiocca, E. A., additional, Godlewski, J., additional, Brown, K., additional, Kwatra, M., additional, Bui, T., additional, Zhu, S., additional, Kozono, D., additional, Li, J., additional, Kushwaha, D., additional, Carter, B., additional, Chen, C., additional, Schulte, J., additional, Srikanth, M., additional, Das, S., additional, Zhang, J., additional, Lathia, J., additional, Yin, L., additional, Rich, J., additional, Olson, E., additional, Kessler, J., additional, Chenn, A., additional, Cherry, A., additional, Haas, B., additional, Lin, Y. H., additional, Ong, S.-E., additional, Stella, N., additional, Cifarelli, C. P., additional, Griffin, R. J., additional, Cong, D., additional, Zhu, W., additional, Shi, Y., additional, Clark, P., additional, Kuo, J., additional, Hu, S., additional, Sun, D., additional, Bookland, M., additional, Darbinian, N., additional, Dey, A., additional, Robitaille, M., additional, Remke, M., additional, Faury, D., additional, Maier, C., additional, Malhotra, A., additional, Jabado, N., additional, Taylor, M., additional, Angers, S., additional, Kenney, A., additional, Ren, X., additional, Zhou, H., additional, Schur, M., additional, Baweja, A., additional, Singh, M., additional, Erdreich-Epstein, A., additional, Fu, J., additional, Koul, D., additional, Yao, J., additional, Saito, N., additional, Zheng, S., additional, Verhaak, R., additional, Lu, Z., additional, Yung, W. K. A., additional, Gomez, G., additional, Volinia, S., additional, Croce, C., additional, Brennan, C., additional, Cavenee, W., additional, Furnari, F., additional, Lopez, S. G., additional, Qu, D., additional, Petritsch, C., additional, Gonzalez-Huarriz, M., additional, Aldave, G., additional, Ravi, D., additional, Rubio, A., additional, Diez-Valle, R., additional, Marigil, M., additional, Jauregi, P., additional, Vera, B., additional, Rocha, A. A. d. l., additional, Tejada-Solis, S., additional, Alonso, M. M., additional, Gopal, U., additional, Isaacs, J., additional, Gruber-Olipitz, M., additional, Dabral, S., additional, Ramkissoon, S., additional, Kung, A., additional, Pak, E., additional, Chung, J., additional, Theisen, M., additional, Sun, Y., additional, Monrose, V., additional, Franchetti, Y., additional, Shulman, D., additional, Redjal, N., additional, Tabak, B., additional, Beroukhim, R., additional, Zhao, J., additional, Buonamici, S., additional, Ligon, K., additional, Kelleher, J., additional, Segal, R., additional, Canton, D., additional, Diaz, P., additional, Scott, J., additional, Hara, K., additional, Kageji, T., additional, Mizobuchi, Y., additional, Kitazato, K., additional, Okazaki, T., additional, Fujihara, T., additional, Nakajima, K., additional, Mure, H., additional, Kuwayama, K., additional, Hara, T., additional, Nagahiro, S., additional, Hill, L., additional, Botfield, H., additional, Hossain-Ibrahim, K., additional, Logan, A., additional, Cruickshank, G., additional, Liu, Y., additional, Gilbert, M., additional, Kyprianou, N., additional, Rangnekar, V., additional, Horbinski, C., additional, Hu, Y., additional, Vo, C., additional, Li, Z., additional, Ke, C., additional, Ru, N., additional, Hess, K. R., additional, Linskey, M. E., additional, Zhou, Y.-a. H., additional, Hu, F., additional, Vinnakota, K., additional, Wolf, S., additional, Kettenmann, H., additional, Jackson, P. J., additional, Larson, J. D., additional, Beckmann, D. A., additional, Moriarity, B. S., additional, Largaespada, D. A., additional, Jalali, S., additional, Agnihotri, S., additional, Singh, S., additional, Burrell, K., additional, Croul, S., additional, Zadeh, G., additional, Kang, S.-H., additional, Yu, M. O., additional, Song, N.-H., additional, Park, K.-J., additional, Chi, S.-G., additional, Chung, Y.-G., additional, Kim, S. K., additional, Kim, J. W., additional, Kim, J. Y., additional, Kim, J. E., additional, Choi, S. H., additional, Kim, T. M., additional, Lee, S.-H., additional, Kim, S.-K., additional, Park, S.-H., additional, Kim, I. H., additional, Park, C.-K., additional, Jung, H.-W., additional, Koldobskiy, M., additional, Ahmed, I., additional, Ho, G., additional, Snowman, A., additional, Raabe, E., additional, Eberhart, C., additional, Snyder, S., additional, Gugel, I., additional, Bornemann, A., additional, Pantazis, G., additional, Mack, S., additional, Shih, D., additional, Sabha, N., additional, Tatagiba, M., additional, Krischek, B., additional, Schulte, A., additional, Liffers, K., additional, Kathagen, A., additional, Riethdorf, S., additional, Westphal, M., additional, Lamszus, K., additional, Lee, J. S., additional, Xiao, J., additional, Patel, P., additional, Schade, J., additional, Wang, J., additional, Deneen, B., additional, Song, H.-R., additional, Leiss, L., additional, Gjerde, C., additional, Saed, H., additional, Rahman, A., additional, Lellahi, M., additional, Enger, P. O., additional, Leung, R., additional, Gil, O., additional, Lei, L., additional, Canoll, P., additional, Sun, S., additional, Lee, D., additional, Ho, A. S. W., additional, Pu, J. K. S., additional, Zhang, X.-q., additional, Lee, N. P., additional, Dat, P. J. R., additional, Leung, G. K. K., additional, Loetsch, D., additional, Steiner, E., additional, Holzmann, K., additional, Pirker, C., additional, Hlavaty, J., additional, Petznek, H., additional, Hegedus, B., additional, Garay, T., additional, Mohr, T., additional, Sommergruber, W., additional, Lukiw, W. J., additional, Jones, B. M., additional, Zhao, Y., additional, Bhattacharjee, S., additional, Culicchia, F., additional, Magnus, N., additional, Garnier, D., additional, Meehan, B., additional, McGraw, S., additional, Hashemi, M., additional, Lee, T. H., additional, Milsom, C., additional, Gerges, N., additional, Trasler, J., additional, Pawlinski, R., additional, Mackman, N., additional, Rak, J., additional, Maherally, Z., additional, Thorne, A., additional, An, Q., additional, Barbu, E., additional, Fillmore, H., additional, Pilkington, G., additional, Tan, S. L., additional, Tan, S., additional, Choi, S., additional, Potts, C., additional, Ford, D. A., additional, Nahle, Z., additional, Kenney, A. M., additional, Matlaf, L., additional, Khan, S., additional, Zider, A., additional, Singer, E., additional, Cobbs, C., additional, Soroceanu, L., additional, McFarland, B. C., additional, Hong, S. W., additional, Rajbhandari, R., additional, Twitty, G. B., additional, Gray, G. K., additional, Yu, H., additional, Benveniste, E. N., additional, Nozell, S. E., additional, Minata, M., additional, Kim, S., additional, Mao, P., additional, Kaushal, J., additional, Nakano, I., additional, Mizowaki, T., additional, Sasayama, T., additional, Tanaka, K., additional, Mizukawa, K., additional, Nishihara, M., additional, Nakamizo, S., additional, Tanaka, H., additional, Kohta, M., additional, Hosoda, K., additional, Kohmura, E., additional, Moeckel, S., additional, Meyer, K., additional, Leukel, P., additional, Bogdahn, U., additional, Riehmenschneider, M. J., additional, Bosserhoff, A. K., additional, Spang, R., additional, Hau, P., additional, Mukasa, A., additional, Watanabe, A., additional, Ogiwara, H., additional, Aburatani, H., additional, Mukherjee, J., additional, Obha, S., additional, See, W., additional, Pieper, R., additional, Otsuka, R., additional, Kung, D., additional, Sinha, T., additional, Meares, G., additional, Nozell, S., additional, Ott, M., additional, Litzenburger, U., additional, Rauschenbach, K., additional, Bunse, L., additional, Pusch, S., additional, Ochs, K., additional, Sahm, F., additional, Opitz, C., additional, von Deimling, A., additional, Wick, W., additional, Platten, M., additional, Peruzzi, P., additional, Read, R., additional, Fenton, T., additional, Wykosky, J., additional, Vandenberg, S., additional, Babic, I., additional, Iwanami, A., additional, Yang, H., additional, Mischel, P., additional, Thomas, J., additional, Ronellenfitsch, M. W., additional, Thiepold, A. L., additional, Harter, P. N., additional, Mittelbronn, M., additional, Steinbach, J. P., additional, Rybakova, Y., additional, Kalen, A., additional, Sarsour, E., additional, Goswami, P., additional, Silber, J., additional, Harinath, G., additional, Aldaz, B., additional, Fabius, A. W. M., additional, Turcan, S., additional, Chan, T. A., additional, Huse, J. T., additional, Sonabend, A. M., additional, Bansal, M., additional, Guarnieri, P., additional, Soderquist, C., additional, Yun, J., additional, Kennedy, B., additional, Sisti, J., additional, Bruce, S., additional, Bruce, R., additional, Shakya, R., additional, Ludwig, T., additional, Rosenfeld, S., additional, Sims, P. A., additional, Bruce, J. N., additional, Califano, A., additional, Stockhausen, M.-T., additional, Kristoffersen, K., additional, Olsen, L. S., additional, Poulsen, H. S., additional, Stringer, B., additional, Day, B., additional, Barry, G., additional, Piper, M., additional, Jamieson, P., additional, Ensbey, K., additional, Bruce, Z., additional, Richards, L., additional, Boyd, A., additional, Sufit, A., additional, Burleson, T., additional, Le, J. P., additional, Keating, A. K., additional, Sundstrom, T., additional, Varughese, J. K., additional, Harter, P., additional, Prestegarden, L., additional, Petersen, K., additional, Azuaje, F., additional, Tepper, C., additional, Ingham, E., additional, Even, L., additional, Johnson, S., additional, Skaftnesmo, K. O., additional, Lund-Johansen, M., additional, Bjerkvig, R., additional, Ferrara, K., additional, Thorsen, F., additional, Takeshima, H., additional, Yamashita, S., additional, Yokogami, K., additional, Mizuguchi, S., additional, Nakamura, H., additional, Kuratsu, J., additional, Fukushima, T., additional, Morishita, K., additional, Tang, Y., additional, Vaka, D., additional, Chen, S., additional, Ponnuswami, A., additional, Cho, Y.-J., additional, Monje, M., additional, Nakamura, T., additional, Cahill, D., additional, Tiemann, K., additional, Hedman, H., additional, Niclou, S. P., additional, Timmer, M., additional, Tjiong, R., additional, Rohn, G., additional, Goldbrunner, R., additional, Stavrinou, P., additional, Perrech, M., additional, Tokita, M., additional, Mikheev, S., additional, Sellers, D., additional, Mikheev, A., additional, Kosai, Y., additional, Rostomily, R., additional, Tritschler, I., additional, Seystahl, K., additional, Schroeder, J. J., additional, Weller, M., additional, Wade, A., additional, Robinson, A. E., additional, Phillips, J. J., additional, Gong, Y., additional, Ma, Y., additional, Cheng, Z., additional, Thompson, R., additional, Fan, Q.-W., additional, Cheng, C., additional, Gustafson, W., additional, Charron, E., additional, Zipper, P., additional, Wong, R., additional, Chen, J., additional, Lau, J., additional, Knobbe-Thosen, C., additional, Jura, N., additional, Reifenberger, G., additional, Shokat, K., additional, Weiss, W., additional, Wu, S., additional, Hu, J., additional, Taylor, T., additional, Villa, G. R., additional, Mischel, P. S., additional, Gonias, S. L., additional, Yamashita, D., additional, Kondo, T., additional, Takahashi, H., additional, Inoue, A., additional, Kohno, S., additional, Harada, H., additional, Ohue, S., additional, Ohnishi, T., additional, Li, P., additional, Ng, J., additional, Yuelling, L., additional, Du, F., additional, Curran, T., additional, Yang, Z.-j., additional, Zhu, D., additional, Castellino, R. C., additional, Van Meir, E. G., additional, Begum, G., additional, Wang, Q., additional, Yang, S.-S., additional, Lin, S.-H., additional, and Kahle, K., additional

Published
2013
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29. HBV/ HIV coinfection is associated with poorer outcomes in hospitalized patients with HBV or HIV.

Author

Rajbhandari, R., Jun, T., Khalili, H., Chung, R. T., and Ananthakrishnan, A. N.

Subjects
*HIV infections, *PORTAL hypertension, *HOSPITAL admission & discharge, *LOGISTIC regression analysis, *HOSPITAL care
Abstract

We examined the impact of HBV/ HIV coinfection on outcomes in hospitalized patients compared to those with HBV or HIV monoinfection. Using the 2011 US Nationwide Inpatient Sample, we identified patients who had been hospitalized with HBV or HIV monoinfection or HBV/ HIV coinfection using ICD-9- CM codes. We compared liver-related admissions between the three groups. Multivariable logistic regression was performed to identify independent predictors of in-hospital mortality, length of stay and total charges. A total of 72 584 discharges with HBV monoinfection, 133 880 discharges with HIV monoinfection and 8156 discharges with HBV/ HIV coinfection were included. HBV/ HIV coinfection was associated with higher mortality compared to HBV monoinfection ( OR 1.67, 95% CI 1.30-2.15) but not when compared to HIV monoinfection ( OR 1.22, 95% CI 0.96-1.54). However, the presence of HBV along with cirrhosis or complications of portal hypertension was associated with three times greater in-hospital mortality in patients with HIV compared to those without these complications ( OR 3.00, 95% CI 1.80-5.02). Length of stay and total hospitalization charges were greater in the HBV-/ HIV-coinfected group compared to the HBV monoinfection group (+1.53 days, P < 0.001; $17595, P < 0.001) and the HIV monoinfection group (+0.62 days, P = 0.034; $8840, P = 0.005). In conclusion, HBV/ HIV coinfection is a risk factor for in-hospital mortality, particularly in liver-related admissions, compared to HBV monoinfection. Overall healthcare utilization from HBV/ HIV coinfection is also higher than for either infection alone and higher than the national average for all hospitalizations, thus emphasizing the healthcare burden from these illnesses. [ABSTRACT FROM AUTHOR]

Published
2016
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30. LAB-CELL BIOLOGY AND SIGNALING

Author

Kijima, N., primary, Hosen, N., additional, Kagawa, N., additional, Hashimoto, N., additional, Chiba, Y., additional, Kinoshita, M., additional, Sugiyama, H., additional, Yoshimine, T., additional, Kim, Y. Z., additional, Kim, K. H., additional, Lee, E. H., additional, Hu, B., additional, Sim, H., additional, Mohan, N., additional, Agudelo-Garcia, P., additional, Nuovo, G., additional, Cole, S., additional, Viapiano, M. S., additional, McFarland, B. C., additional, Hong, S. W., additional, Rajbhandari, R., additional, Twitty, G. B., additional, Kenneth Gray, G., additional, Yu, H., additional, Langford, C. P., additional, Yancey Gillespie, G., additional, Benveniste, E. N., additional, Nozell, S. E., additional, Nitta, R., additional, Mitra, S., additional, Bui, T., additional, Li, G., additional, Munoz, J. L., additional, Rodriguez-Cruz, V., additional, Rameshwar, P., additional, See, W. L., additional, Mukherjee, J., additional, Shannon, K. M., additional, Pieper, R. O., additional, Floyd, D. H., additional, Xiao, A., additional, Purow, B. W., additional, Lavon, I., additional, Zrihan, D., additional, Refael, M., additional, Bier, A., additional, Canello, T., additional, Siegal, T., additional, Granit, A., additional, Xie, Q., additional, Wang, X., additional, Gong, Y., additional, Mao, Y., additional, Chen, X., additional, Zhou, L., additional, Lee, S. X., additional, Tunkyi, A., additional, Wong, E. T., additional, Swanson, K. D., additional, Zhang, K., additional, Chen, L., additional, Zhang, J., additional, Shi, Z., additional, Han, L., additional, Pu, P., additional, Kang, C., additional, Cho, W. H., additional, Ogawa, D., additional, Godlewski, J., additional, Bronisz, A., additional, Antonio Chiocca, E., additional, Mustafa, D. A. M., additional, Sieuwerts, A. M., additional, Smid, M., additional, de Weerd, V., additional, Martens, J. W., additional, Foekens, J. A., additional, Kros, J. M., additional, McCulloch, C., additional, Graff, J., additional, Sui, Y., additional, Dinn, S., additional, Huang, Y., additional, Li, Q., additional, Fiona, G., additional, Nakashima, H., additional, Leiss, L., additional, Manini, I., additional, Enger, P. O., additional, Yang, C., additional, Iyer, R., additional, Yu, A. C. H., additional, Li, S., additional, Ikejiri, B. L., additional, Zhuang, Z., additional, Lonser, R., additional, Massoud, T. F., additional, Paulmurugan, R., additional, Gambhir, S. S., additional, Merrill, M. J., additional, Sun, M., additional, Chen, M., additional, Edwards, N. A., additional, Shively, S. B., additional, Lonser, R. R., additional, Baia, G. S., additional, Caballero, O. L., additional, Orr, B. A., additional, Lal, A., additional, Ho, J. S., additional, Cowdrey, C., additional, Tihan, T., additional, Mawrin, C., additional, Riggins, G. J., additional, Lu, D., additional, Leo, C., additional, Wheeler, H., additional, McDonald, K., additional, Schulte, A., additional, Zapf, S., additional, Stoupiec, M., additional, Kolbe, K., additional, Riethdorf, S., additional, Westphal, M., additional, Lamszus, K., additional, Timmer, M., additional, Rohn, G., additional, Koch, A., additional, Goldbrunner, R., additional, Ruggieri, R., additional, Vanan, I., additional, Dong, Z., additional, Sarkaria, J. N., additional, Tran, N. L., additional, Berens, M. E., additional, Symons, M., additional, Rowther, F. B., additional, Dawson, T., additional, Ashton, K., additional, Darling, J., additional, Warr, T., additional, Okamoto, M., additional, Palanichamy, K., additional, Gordon, N., additional, Patel, D., additional, Walston, S., additional, Krishanan, T., additional, Chakravarti, A., additional, Kalinina, J., additional, Carroll, A., additional, Wang, L., additional, Yu, Q., additional, Mancheno, D. E., additional, Wu, S., additional, Liu, F., additional, Ahn, J., additional, He, M., additional, Mao, H., additional, Van Meir, E. G., additional, Debinski, W., additional, Gonzales, O., additional, Beauchamp, A., additional, Gibo, D. M., additional, Seals, D. F., additional, Speranza, M. C., additional, Frattini, V., additional, Kapetis, D., additional, Pisati, F., additional, Eoli, M., additional, Pellegatta, S., additional, Finocchiaro, G., additional, Maherally, Z., additional, Smith, J. R., additional, Pilkington, G. J., additional, Zhu, W., additional, Wang, Q., additional, Clark, P. A., additional, Yang, S.-S., additional, Lin, S.-H., additional, Kahle, K. T., additional, Kuo, J. S., additional, Sun, D., additional, Hossain, M. B., additional, Cortes-Santiago, N., additional, Gururaj, A., additional, Thomas, J., additional, Gabrusiewicz, K., additional, Gumin, J., additional, Xipell, E., additional, Lang, F., additional, Fueyo, J., additional, Yung, W. K. A., additional, Gomez-Manzano, C., additional, Cook, N. J., additional, Lawrence, J. E., additional, Rovin, R. A., additional, Belton, R. J., additional, Winn, R. J., additional, Ferluga, S., additional, Lee, S.-H., additional, Khwaja, F. W., additional, Zerrouqi, A., additional, Devi, N. S., additional, Drucker, K. L., additional, Lee, H. K., additional, Finniss, S., additional, Cazacu, S., additional, Poisson, L., additional, Xiang, C., additional, Rempel, S. A., additional, Mikkelsen, T., additional, Brodie, C., additional, Shen, J., additional, Kenchappa, R. S., additional, Valadez, J. G., additional, Cooper, M. K., additional, Carter, B. D., additional, Forsyth, P. A., additional, Lee, J. S., additional, Erdreich-Epstein, A., additional, Song, H.-R., additional, Lawn, S., additional, Kenchappa, R., additional, Forsyth, P., additional, Lim, K. J., additional, Bar, E. E., additional, Eberhart, C. G., additional, Blough, M., additional, Alnajjar, M., additional, Chesnelong, C., additional, Weiss, S., additional, Chan, J., additional, Cairncross, G., additional, Wykosky, J., additional, Cavenee, W., additional, Furnari, F., additional, Brown, K. E., additional, Keir, S. T., additional, Sampson, J. H., additional, Bigner, D. D., additional, Kwatra, M. M., additional, Kotipatruni, R. P., additional, Thotala, D. K., additional, Jaboin, J., additional, Taylor, T. E., additional, Schinzel, A. C., additional, Hahn, W. C., additional, Cavenee, W. K., additional, Furnari, F. B., additional, Kapoor, G. S., additional, Macyszyn, L., additional, Bi, Y., additional, Fetting, H., additional, Poptani, H., additional, Ittyerah, R., additional, Davuluri, R. V., additional, O'Rourke, D., additional, Pitter, K. L., additional, Hosni-Ahmed, A., additional, Colevas, K., additional, Holland, E. C., additional, Jones, T. S., additional, Malhotra, A., additional, Potts, C., additional, Fernandez-Lopez, A., additional, Kenney, A. M., additional, Cheng, S., additional, Feng, H., additional, Jarzynka, M. J., additional, Li, Y., additional, Keezer, S., additional, Johns, T. G., additional, Hamilton, R. L., additional, Vuori, K., additional, Nishikawa, R., additional, Fenton, T., additional, Cheng, T., additional, Mikheev, A. M., additional, Mikheeva, S. A., additional, Silber, J. R., additional, Horner, P. J., additional, Rostomily, R., additional, Henson, E. S., additional, Brown, M., additional, Eisenstat, D. D., additional, Gibson, S. B., additional, Price, R. L., additional, Song, J., additional, Bingmer, K., additional, Oglesbee, M., additional, Cook, C., additional, Kwon, C.-H., additional, Nguyen, T. T., additional, Chiocca, E. A., additional, Lukiw, W. J., additional, Culicchia, F., additional, Jones, B. M., additional, Zhao, Y., additional, and Bhattacharjee, S., additional

Published
2012
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34. Projected changes in climate over the Indus river basin using a high resolution regional climate model (PRECIS).

Author

Rajbhandari, R., Shrestha, A., Kulkarni, A., Patwardhan, S., and Bajracharya, S.

Subjects
*CLIMATE change, *ATMOSPHERIC models, *PREDICTION models, *METEOROLOGICAL precipitation
Abstract

A regional climate modelling system, the Providing REgional Climates for Impacts Studies developed by the Hadley Centre for Climate Prediction and Research, has been used to study future climate change scenarios over Indus basin for the impact assessment. In this paper we have examined the three Quantifying Uncertainty in Model Predictions simulations selected from 17-member perturbed physics ensemble generated using Hadley Centre Coupled Module. The climate projections based on IPCC SRES A1B scenario are analysed over three time slices, near future (2011-2040), middle of the twenty first century (2041-2070), and distant future (2071-2098). The baseline simulation (1961-1990) was evaluated with observed data for seasonal and spatial patterns and biases. The model was able to resolve features on finer spatial scales and depict seasonal variations reasonably well, although there were quantitative biases. The model simulations suggest a non-uniform change in precipitation overall, with an increase in precipitation over the upper Indus basin and decrease over the lower Indus basin, and little change in the border area between the upper and lower Indus basins. A decrease in winter precipitation is projected, particularly over the southern part of the basin. Projections indicate greater warming in the upper than the lower Indus, and greater warming in winter than in the other seasons. The simulations suggest an overall increase in the number of rainy days over the basin, but a decrease in the number of rainy days accompanied by an increase in rainfall intensity in the border area between the upper and lower basins, where the rainfall amount is highest. [ABSTRACT FROM AUTHOR]

Published
2015
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35. The National Institute of Health Stroke Scale Score and Outcome in Acute Ischemic Stroke.

Author

Gajurel, B. P., Dhungana, K., Parajuli, P., Karn, R., Rajbhandari, R., Kafle, D., and Oli, K. K.

Subjects
STROKE, ISCHEMIA, CENTRAL nervous system, HEALTH outcome assessment, COMPUTED tomography, PATIENTS
Abstract

Introduction: Stroke is a focal neurological deficit of sudden onset which lasts for more than 24 hours and has a vascular cause. Various prognostic indices derived from clinical features or patient characteristics and ancillary tests have been used to predict the prognosis of patients with acute ischemic stroke. The aim of this study was to find out the significance of the National Institute of Health Stroke Scale (NIHSS) score on admission in predicting the prognosis of patients with acute ischemic stroke. Method: This is a prospective observational study done in a tertiary care hospital with two hundred patients with acute ischemic stroke Result: Themean NIHSS score in patients with better outcome was 4.6 (? 2.2) and the mean NIHSS score in patients with poor outcome was 14.16(? 7.96). The difference was statistically significant (p=0.000). Conclusion: The baseline neurological status as measured by the National Institute of Health Stroke Scale score predicts the functional status at one month after acute ischemic stroke. [ABSTRACT FROM AUTHOR]

Published
2014
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38. COMPARISION OF TRANSTHORACIC AND TRANSESOPHAGEAL ECHOCARDIOGRAPHY FOR THE DETECTION OF LEFT ATRIAL THROMBUS IN RHEUMATIC MITRAL VALVULAR DISEASE.

Author

Sharma, D., Maskey, A., KC, M. B., Rajbhandari, S., Rajbhandari, R., Limbu, Y. R., Malla, R., Regmi, S. R., Pradhan, B., and Shrestha, N. K.

Abstract

Transesophageal echocardiography (TEE) was performed in 114 patients with rheumatic mitral valvular disease and its results were compared with those of transthoracic echocardiography (TTE). Of 114 patients, LA thrombus was detected in 32 patients by TEE whereas TTE could detect LA thrombus in only 4 of them. Out of 32 cases, thrombus was located in left atrial appendage (LAA) in 20 (62%). TTE could detect LAA thrombus in only one patient. The procedure of TEE was well tolerated and there was no complication. We conclude that TEE is superior to TTE in detection of LA cavity and LA appendage thrombus. Hence, TEE should be a routine procedure to detect LA thrombus in patients with rheumatic mitral valvular disease prior to any surgical intervention. [ABSTRACT FROM AUTHOR]

Published
2004
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40. Crossing the quality chasm in resource-limited settings

Author

Maru Duncan, Andrews Jason, Schwarz Dan, Schwarz Ryan, Acharya Bibhav, Ramaiya Astha, Karelas Gregory, Rajbhandari Ruma, Mate Kedar, and Shilpakar Sona

Subjects
Resource-limited, Health system, Global health, Quality improvement, Public aspects of medicine, RA1-1270
Abstract

Abstract Over the last decade, extensive scientific and policy innovations have begun to reduce the “quality chasm” - the gulf between best practices and actual implementation that exists in resource-rich medical settings. While limited data exist, this chasm is likely to be equally acute and deadly in resource-limited areas. While health systems have begun to be scaled up in impoverished areas, scale-up is just the foundation necessary to deliver effective healthcare to the poor. This perspective piece describes a vision for a global quality improvement movement in resource-limited areas. The following action items are a first step toward achieving this vision: 1) revise global health investment mechanisms to value quality; 2) enhance human resources for improving health systems quality; 3) scale up data capacity; 4) deepen community accountability and engagement initiatives; 5) implement evidence-based quality improvement programs; 6) develop an implementation science research agenda.

Published
2012
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41. A study of the prevalence and risk factors leading to HIV infection among a sample of street children and youth of Kathmandu

Author

Karmacharya Dibesh, Yu Dongmei, Dixit Sameer, Rajbhandari Rajesh, Subedi Bhawana, Shrestha Sonu, Manandhar Sulochana, and Santangelo Susan L

Subjects
HIV prevalence, Behavioural risk factors, Intravenous drug use, Street children, Kathmandu, Nepal, Immunologic diseases. Allergy, RC581-607
Abstract

Abstract Background The true prevalence of HIV and other sexually transmitted diseases among street children in Nepal is virtually unknown while information on related behavioural risk factors in this population is non-existent. The risk of HIV infection among street children and adolescents may be especially high due to their marginalized social and economic conditions. This study was conducted to determine the prevalence of HIV infection among a sample of street children and youth of Kathmandu and to identify risk factors associated with HIV infection in this group. A sample of street children and youth was recruited based on the purposive sampling of ten streets in Kathmandu, Nepal, known to have a high density of street children and youth. A total of 251 street children (aged 11–16 years) and youth (aged 17–24 years) were enrolled, with informed consent, from November, 2008 through June, 2009. Most of the participants (95%) were male. Case status was determined by serological assessment of HIV status; data on risk factors were obtained using structured survey interviews. HIV prevalence and rates of a number of behavioural risk factors suspected to play a role in HIV transmission among street children and youth were determined, including unprotected sex, intravenous drug use, and other risky sex and substance use behaviours. Results Among the 251 children and youth, we found an overall HIV prevalence of 7.6%. As the sample size of females was small (n = 13) and the behavioural risk factors are likely to be quite different for boys and girls, we conducted separate analyses by gender. As our small sample of females is unlikely to be representative and lacks power for statistical testing, our report focuses on the results for the males surveyed.The strongest behavioural risk factor to emerge from this study was intravenous drug use; 30% of the male subjects were injecting drug users and 20% of those were HIV positive. Furthermore, frequency of drug injection was a highly significant predictor with a dose–response relationship; males reporting occasional injection drug use were nearly 9 times more likely to be HIV positive than never users, while weekly drug injectors had over 46 times the risk of non-users, controlling for exposure to group sex, the only other significant risk factor in the multivariate model. Conclusions This sample of street children and youth of Kathmandu has a nearly 20-fold higher prevalence of HIV infection than the general population of Nepal (0.39%). The children and youth engage in number of high risk behaviours, including intravenous drug use, putting them at significant risk of contracting HIV and other sexually transmitted infections.

Published
2012
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42. Segmental resection with primary reconstruction using patient- specific implant for odontogenic fibromyxoma: An illustrative rare case from Nepal.

Author

Ghimire B, Miya N, Rajbhandari R, and Verma L

Abstract

Introduction: Odontogenic fibromyxoma (OFM) is a round and locally invasive neoplasm predominantly seen in the mandible. Though radiographic appearance is variable, definitive diagnosis is based on correlation with histopathological examination. Surgical approach is the treatment of choice. For reconstruction, patient-specific implant (PSI) has lately been developed as a crucial help., Case Presentation: This case report presents a 19 year old female patient with odontogenic fibromyxoma highlighting its clinical, radiographic, histopathological features along with rehabilitation using patient specific implants reducing the complexity and related morbidities of reconstructive procedures., Discussion: Surgical repair and reconstruction of defects in cranio-maxillofacial region is challenging. The described treatment eliminates the need for bone grafting, shows optimal results owing to the shorter rehabilitation time and more accurate fits., Conclusion: This report introduces a novel technique whereby patient-specific implants are employed as the primary method of reconstruction following segmental resection., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interest or personal relationship that could have appeared to influence the work reported in this paper., (Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Published
2024
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43. Changes in the Prevalence, Incidence, and Disability-Adjusted Life Years of Non-alcoholic Fatty Liver Disease in the United States Between 1990 and 2019.

Author

Jaroenlapnopparat A, Mettler SK, Guillen H, Chayanupatkul M, and Rajbhandari R

Subjects
United States epidemiology, Humans, Child, Middle Aged, Child, Preschool, Adolescent, Young Adult, Adult, Disability-Adjusted Life Years, Prevalence, Incidence, Massachusetts, Quality-Adjusted Life Years, Global Health, Non-alcoholic Fatty Liver Disease diagnosis, Non-alcoholic Fatty Liver Disease epidemiology
Abstract

Introduction: This study aimed to determine trends in the prevalence, incidence, and disability-adjusted life years (DALYs) of Non-alcoholic Fatty Liver Disease (NAFLD) in the US across different states and age groups between 1990 and 2019., Methods: Using the Global Burden of Disease database, this study analyzed the prevalence, incidence, and DALYs of NAFLD in the US between 1990 and 2019. We computed relative percentage changes, performed Joinpoint regression analyses of trends, and compared these between states and age groups (5-19, 20-55, and more than 55 years old)., Results: In the United States, the prevalence of NAFLD increased more than the global average over the study period (+ 30.7% vs. + 24.5%), especially in the 5-19-year-old age group. Among all states, Kansas, Washington, and California had the highest increase in prevalence and the District of Columbia followed by Massachusetts and North Carolina had the lowest increase in prevalence. The increase in incidence was greater in the US than the global average (+ 37.18% vs. + 7.28%). West Virginia, Ohio, and Kentucky had the highest increase in incidence. The increase in DALYs was greater in the US compared to the global average (+ 57.15% vs. + 12.65%). Alaska, West Virginia, and Kentucky had the highest increase in DALYs. The increased incidence and DALYs were found in all states except in the District of Columbia., Conclusion: The prevalence of NAFLD in the US has increased more rapidly than the global average, especially in the pediatric population. South and Midwest states have the highest increase in prevalence, incidence, and DALYs of NAFLD. The District of Columbia was the only state that has decreased incidence and DALYs., (© 2024. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.)

Published
2024
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45. SOFA and APACHE II scoring systems for predicting outcome of neurological patients admitted in a tertiary hospital intensive care unit.

Author

Shahi S, Paneru H, Ojha R, Karn R, Rajbhandari R, and Gajurel BP

Abstract

Background: The field of neurology encompasses the study and treatment of disorders that affect the nervous system, and patients with neurological conditions often require specialized care, particularly in the ICU. Predictive scoring systems are measures of disease severity used to predict patient outcomes. The aim of this study was to compare the discriminative power of commonly used scoring systems, namely the sequential organ failure assessment (SOFA) and acute physiology and chronic health evaluation II (APACHE II) in the ICU of a tertiary care hospital., Methods: This retrospective study included patients with neurological disorders in the ICUs of Tribhuvan University Teaching Hospital from 1 January 2022 to 31 December 2022., Results: A total of 153 patients were included. The mean age of the patients was 54.76 ± 17.32 years with higher male predominance (60.78%). Ischaemic stroke was the most common neurological disorder. There were 58 patients (37.9%) who required mechanical ventilation and all-cause mortality was 20.9%. The mean SOFA score was significantly higher ( P =0.002) in survivors, whereas the mean APACHE II did not show a significant difference ( P =0.238). Receiver operating characteristic (ROC) analysis showed the area of curve (AUC) of SOFA score was 0.765 and of APACHE II was 0.722., Conclusions: SOFA score had comparatively higher discriminative power than APACHE II. Assessment of the performance of scoring systems in a specific ICU setting improves the sensitivity and applicability of the model to these settings., Competing Interests: There are no conflicts of interest.Sponsorships or competing interests that may be relevant to content are disclosed at the end of this article., (Copyright © 2024 The Author(s). Published by Wolters Kluwer Health, Inc.)

Published
2024
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46. Dyslipidemia and Obesity in Ischemic Stroke.

Author

Gajurel BP, Gurung A, Ojha R, Rajbhandari R, and Karn R

Abstract

Background Dyslipidemia and obesity are both important risk factors for the first and recurrent ischemic strokes. Dyslipidemia is highly prevalent among Asian populations, and the prevalence of obesity is also noted to be progressively increasing in this population. This study was carried out to determine the prevalence of dyslipidemia and central obesity and their association with each other and various cardiovascular risk factors among patients who presented with an acute ischemic stroke to a tertiary care university hospital in Nepal. Methods This study is a secondary analysis done on data from a prospective observational study that was carried out on patients who were either acutely admitted to or visited the outpatient department of the hospital with a diagnosis of ischemic stroke. Dyslipidemia was defined according to the third report of the National Cholesterol Education Program expert panel on detection, evaluation, and treatment of high blood cholesterol in adults. Obesity was defined as central obesity by measuring waist circumference. Data were collected by convenience sampling and analyzed by IBM Corp. Released 2019. IBM SPSS Statistics for Windows, Version 26.0. Armonk, NY: IBM Corp. Significant variables were compared with logistic regression analysis. Other data were expressed as frequencies and percentages. Results Out of 145 patients, 77 were male (53.1%). The mean age of the patients was 60.15 years. Dyslipidemia and central obesity were present in 96.6% and 57.9% of the patients, respectively. The most common lipid abnormality was low-density lipoprotein cholesterol, present in 82.8% of the patients, followed by high triglycerides, present in 21.4% of them. Dyslipidemia was not associated with any vascular risk factors. Central obesity was significantly associated with female gender, diabetes, and low-density lipoprotein cholesterol in univariate analysis. However, in multivariate logistic regression analysis, it was significantly associated with only female gender (p=0.003) and diabetes (p=0.012). Conclusion Dyslipidemia and central obesity are very common in patients with ischemic stroke. Dyslipidemia is not associated with any vascular risk factors. However, central obesity is significantly associated with the female gender and diabetes., Competing Interests: The authors have declared that no competing interests exist., (Copyright © 2023, Gajurel et al.)

Published
2023
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47. Haploinsufficiency of NFKBIA reshapes the epigenome antipodal to the IDH mutation and imparts disease fate in diffuse gliomas.

Author

Bredel M, Espinosa L, Kim H, Scholtens DM, McElroy JP, Rajbhandari R, Meng W, Kollmeyer TM, Malta TM, Quezada MA, Harsh GR, Lobo-Jarne T, Solé L, Merati A, Nagaraja S, Nair S, White JJ, Thudi NK, Fleming JL, Webb A, Natsume A, Ogawa S, Weber RG, Bertran J, Haque SJ, Hentschel B, Miller CR, Furnari FB, Chan TA, Grosu AL, Weller M, Barnholtz-Sloan JS, Monje M, Noushmehr H, Jenkins RB, Rogers CL, MacDonald DR, Pugh SL, and Chakravarti A

Subjects
Child, Humans, Epigenome, Haploinsufficiency genetics, Mutation genetics, NF-KappaB Inhibitor alpha genetics, Isocitrate Dehydrogenase, Brain Neoplasms genetics, Glioma genetics, Glioma pathology
Abstract

Genetic alterations help predict the clinical behavior of diffuse gliomas, but some variability remains uncorrelated. Here, we demonstrate that haploinsufficient deletions of chromatin-bound tumor suppressor NFKB inhibitor alpha (NFKBIA) display distinct patterns of occurrence in relation to other genetic markers and are disproportionately present at recurrence. NFKBIA haploinsufficiency is associated with unfavorable patient outcomes, independent of genetic and clinicopathologic predictors. NFKBIA deletions reshape the DNA and histone methylome antipodal to the IDH mutation and induce a transcriptome landscape partly reminiscent of H3K27M mutant pediatric gliomas. In IDH mutant gliomas, NFKBIA deletions are common in tumors with a clinical course similar to that of IDH wild-type tumors. An externally validated nomogram model for estimating individual patient survival in IDH mutant gliomas confirms that NFKBIA deletions predict comparatively brief survival. Thus, NFKBIA haploinsufficiency aligns with distinct epigenome changes, portends a poor prognosis, and should be incorporated into models predicting the disease fate of diffuse gliomas., Competing Interests: Declaration of interests M.B.: NIH/NINDS grant R01NS117641, advisory board membership and grant (Varian Medical Systems), consulting (MRIMath). L.E.: Instituto de Salud Carlos III grant PI22/00069. T.L.-J.: an Asociación Española Contra el Cáncer grant. G.R.H.: NIH/NIBIB grant (P41-EB032840-01). J.S.B.-S.: NIH/NCI grants to Case Western Reserve University School of Medicine. J.P.M.: MOU between OSU Radiation Oncology and OSU Center for Biostatistics. R.B.J.: NIH grants. A.W.: MOU between OSU Radiation Oncology and OSU Biomedical Informatics. T.A.C.: grants (PGDX, Pfizer, AstraZeneca, Illumina, NysnoBio); royalties (PGDX); consulting (Illumina, NysnoBio, Pfizer, BMS, Merck, LG Chem); patents (TMB as predictor of immunotherapy response); stock (Griststone Bio, Nysno Bio). A.N.: consulting (Daiichi-Sankyo, NGK, SparkPlug) to his institution; $2,000 as honoraria. M.W.: grants (Apogenix, Merck, Sharp & Dohme, Merck [EMD], Philogen, Quercis); consulting (Adastra, Medac, Merck [EMD], Nerviano Medical Sciences, Novartis); honoraria (Bristol Meyer Squibb, Medac, Merck, Sharp & Dohme); Data Safety Monitoring Board or Advisory Board (Orbus and Philogen); leadership (EORTC). R.G.W.: grant 70-3163-Wi 3 (German Cancer Aid). S.O.: grant (Cordia Therapeutics, Dainippon-Sumitomo, Pharmaceuticals, Otsuka Assay Inc.); royalties (ASAHI Genomics, Qiagen); consulting (Cordia Therapeutics, Kan Research Institute, Novartis Pharmaceuticals); honoraria (MSD Japan, Kyowa Hakko Kirin, Pfizer); stocks (ASAHI Genomics, Cordia Therapeutics, Rebirthell). M.M.: NIH grants (R01NS092597, DP1NS111132, P50CA165962), grants from Kleberg Foundation Cancer Research, Ludwig Fund for Cancer Research; consulting (Cygnal Therapeutics); honoraria for lectures. C.R.M. and F.B.F.: NIH grant (R01CA258248). F.B.F.: NIH grant (R01NS080939). D.R.M.: clinical trials support (INTELLANCE-1 study [M13-813], CCTG CE.8 [EORTC-1709-BTG], INDIGO study [AG-881], Abbvie, Celgene, Agios [now Servier])., (Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.)

Published
2023
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48. Stroke patterns and outcomes during the second wave of COVID-19 pandemic: a cross-sectional study.

Author

Gajurel BP, Giri S, Tamrakar P, Pandeya A, Gautam N, Shrestha A, Karn R, Rajbhandari R, and Ojha R

Abstract

The coronavirus disease 2019 (COVID-19) pandemic has affected the number of stroke activations, admission of patients with various types of strokes, the rate and timely administration of reperfusion therapy, and all types of time-based stroke-related quality assessment metrics. In this study, we describe the different types of strokes, different delays in seeking and completing treatment occurring during the second wave of the COVID-19 pandemic, and predictors of outcome at 3 months follow-up., Materials and Methods: This is a single-centered prospective cross-sectional study carried out from May 2021 to November 2021, enrolling patients with stroke. Data collected were demographic characteristics, stroke types and their outcomes, and different types of prehospital delays., Results: A total of 64 participants were included in the study with a mean age of 60.25±15.31 years. Ischemic stroke was more common than hemorrhagic stroke. The median time of arrival to the emergency room of our center was 24 h. The most common cause of prehospital delay was found to be delays in arranging vehicles. The median duration of hospital stays [odds ratio (OR)=0.72, P <0.05] and baseline NIHSS (National Institute of Health Stroke Scale) score (OR=0.72, P <0.05) were found to be a predictor of good outcomes at 3 months follow-up on binary logistic regression., Conclusion: The factors that cause the delayed transfer to the hospital and onset of treatment should be addressed. Patient counseling about the likely prognosis can be done after evaluating the probable outcome based on the NIHSS score and median duration of hospital stay. Nevertheless, mechanisms should be developed to reduce the prehospital delay at the ground level as well as at the policy level., Competing Interests: No potential conflicts of interest relevant to this article exists., (Copyright © 2023 The Author(s). Published by Wolters Kluwer Health, Inc.)

Published
2023
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49. Painful tonic spasms in a patient with neuromyelitis optica spectrum disorder: A case report.

Author

Aryal R, Homagian S, Shrestha S, Gajurel BP, Karn R, Rajbhandari R, Gautam N, Shrestha A, Shahi S, and Ojha R

Abstract

Painful tonic spasms initially described in association with multiple sclerosis are actually more common in patients with neuromyelitis optica spectrum disorder. Characterized by fierce pain and tonic posture of limbs, painful tonic spasms are common in patients during the recovery phase after the first episode of myelitis. A 68-year-old man presented with painful tonic spasm after 2 months of diagnosis of neuromyelitis optica spectrum disorder. Eventual use of eslicarbazepine resulted in significant control of spasms. Early recognition of painful tonic spasms and appropriate therapeutic medications can significantly decrease the impact it can have on the quality of life among neuromyelitis optica spectrum disorder patients., Competing Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2023.)

Published
2023
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50. Crohn's Disease Among the Poorest Billion: Burden of Crohn's Disease in Low- and Lower-Middle-Income Countries.

Author

Rajbhandari R, Blakemore S, Gupta N, Mannan S, Nikolli K, Yih A, Drown L, and Bukhman G

Subjects
Humans, Cross-Sectional Studies, Developing Countries, Crohn Disease diagnosis, Crohn Disease epidemiology, Crohn Disease therapy, Inflammatory Bowel Diseases epidemiology, Colitis, Ulcerative epidemiology
Abstract

Background: To establish the epidemiology and patterns of care of Crohn's Disease in low- and lower-middle-income countries., Methods: A cross-sectional survey of gastroenterology providers in countries where the world's poorest billion live was conducted to learn more about the state of diagnostic and treatment capacity for Crohn's. Quantitative data were analyzed in R and Excel., Results: A total of 46 survey responses from 15 countries were received, giving a response rate of 54.8%. All responses collected were from providers practicing in Africa and South Asia. The mean number of patients with Crohn's cared for in the last year was 89.5 overall but ranged from 0 reported at one facility in Rwanda to 1000 reported at two different facilities in India. Overall, Crohn's disease made up 20.6% of the inflammatory bowel disease diagnoses reported by survey respondents, with Africa exhibiting a larger proportion of Crohn's compared to ulcerative colitis than Asia. Most providers reported that patients with Crohn's have symptoms for 6-24 months prior to diagnosis and that 26-50% of their patients live in rural areas. The most reported diagnostic challenges are differentiating between Crohn's and intestinal tuberculosis, poor disease awareness, and lack of trained pathologists. The most widely reported challenge in managing Crohn's disease is patients' inability to afford biologics, reported by 65% of providers., Conclusion: Our study suggests there may be a greater burden of Crohn's disease in low- and lower-middle-income countries than is indicated in prior literature. Respondents reported many challenges in diagnosing and treating Crohn's disease., (© 2022. The Author(s).)

Published
2023
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