Use of donated clotting factors for surgeries on haemophilic patients in a resource‐constrained country: a kind donor, good outcome, change of practice and future directions

Poudyal, Bishesh Sharma; Shrestha, Gentle Sunder; Tuladhar, Sampurna; Gyawali, Bishal; Sedain, Gopal; Battajo, R.; Maskey, Pukar; Paudyal, Sanjaya; Regmi, Subodh; Rc, Dhirga Raj; Kouides, Peter A.

doi:10.1111/hae.13017

Cited by 3 publications

(3 citation statements)

References 5 publications

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“…Dr Bishesh Sharma Poudyal talked about the value of mentorship and friendship in developing an academic career. He walked us through his inspiring and unique experience of international mentorship in improving his clinical practice and expanding the horizons of patient care [ 30 ].…”

Section: Highlights Of Daymentioning

confidence: 99%

Highlights from ecancer Choosing Wisely Nepal 2022: critical appraisal skills for evidence-based practice, 24th–25th September 2022, Kathmandu, Nepal

Poudyal¹,

Dulal²,

Shilpakar³

et al. 2022

ecancer

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The ecancer Kathmandu 2022 workshop on the 24th-25th September 2022 was the first ecancer conference organised in Nepal, a Southeast Asian nation sandwiched between India and China. It was focused on critical appraisal skills for evidence-based practice and was organised in partnership with the Karnali Academy of Health Sciences and the Civil Service Hospital from Nepal, and the Queen's Global Oncology Program from Canada. The workshop emphasised the need for critical thinking in understanding clinical research, and also motivated the delegates to undertake meaningful clinical research relevant to the local setting. The sessions highlighted the features of a good clinical research, identify pitfalls in the reporting of clinical trials, implementation of the research into locally relevant practice and development of local clinical guidelines. Furthermore, the faculty also discussed how to write a good scientific paper, the do's and don'ts of a systematic review and meta-analysis, the role of peer-review and how to do one properly and what do editors look for in evaluating papers submitted for publication. The audience learned the importance of finding a good mentor and fostering local and international collaboration. The local faculty also highlighted their own personal journeys and how mentorship and global collaboration played an important role in their own academic career. The enthusiastic panel discussion was a highlight of the programme where the delegates learned about several important topics from the faculties, such as work-life balance, the role of mentorship in building careers and building networks.

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Section: Highlights Of Daymentioning

confidence: 99%

Highlights from ecancer Choosing Wisely Nepal 2022: critical appraisal skills for evidence-based practice, 24th–25th September 2022, Kathmandu, Nepal

Poudyal¹,

Dulal²,

Shilpakar³

et al. 2022

ecancer

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“…Recent outcomes reported from LMIC HTCs reflect the persistently increased morbidity and mortality 71‐79 . In LMIC, the gap in life expectancy of haemophilia patients versus the general population is significantly greater than in HIC with approximately 30%, 64%, 77% and 93% life expectancy disadvantage in HIC, U‐MIC, L‐MIC and LIC, respectively 1,65,66 .…”

Section: Gene Therapy For Haemophilia In Resource‐constrained Countriesmentioning

confidence: 99%

Hemophilia gene therapy—New country initiatives

2020

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Gene therapy is an opportunity for haemophilia patients to receive a one‐time treatment and have lasting factor levels for years or decades instead of dependence on repeated administration within short intervals and on sustained supply of drug. Great strides have been made in the development of gene therapy for haemophilia in the last decade. Adeno‐associated virus (AAV) vector–mediated gene transfer in haemophilia A and B has entered the phase III trial stage. Gene transfer by lentiviral vector or gene editing technologies using factor VIII (FVIII) or IX (FIX) genes are now entering clinical evaluation. It is expected that the first FVIII and FIX gene therapy products will soon be approved and distributed in major markets. Global access to gene therapy is a critical goal. This review presents new and ongoing efforts towards this goal in countries other than North America and Europe. In Japan, researchers, regulators and funders have established a promising gene therapy development platform for multiple diseases including haemophilia. Decades of scientific and clinical research in haemophilia gene therapy in China have led to a recently registered clinical trial of AAV‐mediated gene therapy for haemophilia B. Other countries are in earlier phases of building gene therapy programmes or participate in international trials. A phase 2 feasibility trial of AAV‐mediated FIX gene therapy in low‐ and middle‐income countries aims to demonstrate that gene therapy could become available in resource‐constrained socio‐economic settings. The different strategies for establishing gene therapy provide opportunities for closing the global gap in haemophilia care.

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“…Nepal solely depends on humanitarian aid programmes and donated factor products from organizations such as The World Federation of Hemophilia (WFH) and Save One Life. Eighty per cent of Nepal's haemophilia patients are physically disabled 5 . Patients are treated on demand, and only 2 patients are receiving prophylaxis treatment.…”

mentioning

confidence: 99%