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Pasireotide treatment for severe congenital hyperinsulinism due to a homozygous ABCC8 mutation.
Author: BarthlenWinfried, BikkerHennie, MohnikeKlaus, MooijChristiaan F, OomenMatthijs W N, TackeCarline E, Zwaveling-SoonawalaNitash, van AlbadaMirjam E, van TrotsenburgA S Paul
Original Abstract of the Article :
ABCC8 and KCJN11 mutations cause the most severe diazoxide-resistant forms of congenital hyperinsulinism (CHI). Somatostatin analogues are considered as secondline treatment in diazoxide-unresponsive cases. Current treatment protocols include the first-generation somatostatin analogue octreotide, al...See full text at original site
Dr.Camel's Paper Summary Blogラクダ博士について
ラクダ博士は、Health Journal が論文の内容を分かりやすく解説するために作成した架空のキャラクターです。
難解な医学論文を、専門知識のない方にも理解しやすいように、噛み砕いて説明することを目指しています。
* ラクダ博士による解説は、あくまで論文の要点をまとめたものであり、原論文の完全な代替となるものではありません。詳細な内容については、必ず原論文をご参照ください。
* ラクダ博士は架空のキャラクターであり、実際の医学研究者や医療従事者とは一切関係がありません。
* 解説の内容は Health Journal が独自に解釈・作成したものであり、原論文の著者または出版社の見解を反映するものではありません。
引用元:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8749021/
データ提供:米国国立医学図書館(NLM)
Pasireotide: A New Hope for Severe Congenital Hyperinsulinism?
Congenital hyperinsulinism (CHI), a rare and often life-threatening condition, can lead to severe hypoglycemia. This study reports the first off-label use of pasireotide, a somatostatin analogue, in a boy with a severe and diazoxide-resistant form of CHI. The researchers describe the patient's treatment journey, including partial pancreatectomy and the subsequent initiation of pasireotide therapy. They found that while pasireotide slightly improved glycemic control, it was not sufficient to prevent near-total pancreatectomy. This research highlights the potential of pasireotide as a treatment option for severe CHI, but also underscores the limitations of this medication and the need for further research.
Exploring New Treatment Frontiers
The study's findings offer valuable insights into the potential of pasireotide as a treatment option for severe CHI. While the researchers' experience with this patient indicates that pasireotide may not be a definitive solution, it highlights the need for further investigation into this medication's role in managing this challenging condition. This research encourages the development of new and effective therapies for patients with severe CHI.
Managing Rare Diseases: A Collaborative Effort
Rare diseases pose unique challenges for healthcare providers and researchers. This study highlights the importance of ongoing research and collaboration to develop effective treatments for these conditions. By sharing knowledge and experiences, we can work towards a future where all individuals with rare diseases have access to quality care and effective therapies.
Dr.Camel's Conclusion
Just as a camel's resilience enables it to survive in the harsh desert environment, patients with rare diseases like CHI require persistent exploration and innovative treatment strategies. This research highlights the importance of seeking new therapeutic options and embracing collaboration to improve patient outcomes.
Date :
- Date Completed n.d.
- Date Revised 2023-11-05
Further Info :
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