• About
  • Masthead
  • License Content
  • Advertise
  • Submit Press Release
  • RSS/Email List
  • Write for us
  • Contact us
2 Minute Medicine
No Result
View All Result

No products in the cart.

SUBSCRIBE
  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • AccountLog-in/out
    • Subscribe
    • Sign-in
    • My account
2 Minute Medicine
  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • AccountLog-in/out
    • Subscribe
    • Sign-in
    • My account
SUBSCRIBE
2 Minute Medicine
Subscribe
Home All Specialties Chronic Disease

Recombinant viral vector treatment sustains endogenous factor IX activity in patients with hemophilia B

byShani ChibberandDayton McMillan
December 8, 2017
in Chronic Disease, Infectious Disease, Oncology, Pediatrics
Reading Time: 3 mins read
0
Share on FacebookShare on Twitter

1. A one-time infusion of a recombinant viral vector with a highly expressed factor IX gene resulted in sustained endogenous factor IX coagulant activity among patients with hemophilia B.

2. Transient and asymptomatic increases in alanine aminotransferase (ALT) were observed in study participants.

Evidence Rating Level: 4 (Below Average)

Study Rundown: Hemophilia B is characterized by a deficiency in clotting factor IX and commonly manifests with hemarthrosis. Current treatment includes regularly administered exogenous factor IX, which requires numerous clinical visits and is not always effective for bleeding prevention. This phase 1-2a study assessed the recombinant adeno-associated virus (AAV) vector SPK-9001, which included a gain-of-function variant gene of factor IX (factor-IX-R338L). The primary objective was evaluation of adverse events, with secondary objectives to evaluate factor IX activity. Of 10 patients treated 40 total adverse events were observed, with the most serious being a transient increase in ALT in 1 patient. Factor IX analysis indicated a one-time infusion of SPK-9001 resulted in sustained endogenous factor IX activity, a reduced need for exogenous hemophilia injections, reduced factor IX consumption, and decreased annualized bleeding rates over the cumulative 52 week follow-up period. These results indicate few adverse effects associated with the viral vector used and suggest a useful hemophilia B therapy. Being a phase 1-2a study, a larger follow-up trial is needed to better monitor the safety profile and long-term efficacy of the SPK-9001 vector.

Click to read the study, published in NEJM

Relevant Reading: Adeno-associated viral vectors for the treatment of hemophilia

In-Depth [case series]: This multicenter, nonrandomized, open-label phase 1-2 trial recruited 14 men aged 18 to 53 with hemophilia B and less than 2% of normal factor IX activity. Of those enrolled, 10 men ultimately received a one-time intravenous infusion of the AAV SPK-9001 vector with variant factor-IX-R338L gene. Patients were followed for 492 weeks cumulatively after infusion (mean follow-up time 49±13.4 weeks, range 28 to 78 weeks). There were 40 adverse events reported; none were regarded as serious. A transient elevation in ALT over 2.5 times the upper limit of normal was observed in one patient. Another participant also exhibited increased ALT levels though within the normal range. Other adverse events included upper respiratory infections, gastroesophageal reflux, allergic rhinitis, and arthritis, however these were all deemed to be independent to the study intervention.

RELATED REPORTS

Beremagene geperpavec improves wound healing in dystrophic epidermolysis bullosa

Low-dose AAVS3 gene therapy is efficacious for treating patients with hemophilia B

Valoctocogene roxaparvovec gene therapy efficacious for hemophilia A treatment

Mean (± standard deviation) vector-derived factor IX activity was 33.7±18.5% (range, 14-81) of normal at 52 weeks follow-up. The annualized bleeding rate decreased significantly after vector infusion (mean rate 11.1 events per year before vector infusion [range 0-48 events] vs. 0.4 events per year after administration [range 0-4 events]; p = 0.02). Consumption of factor IX was significantly reduced post-treatment (mean 2908 IU/kg pre-treatment [range 0-8090 IU/kg] vs. 49.3 IU/kg post-treatment [range 0-378 IU/kg]; p = 0.004). Patients required fewer factor IX transfusions after treatment (mean 67.5 [range 0-159] pre-treatment vs. 1.2 [range 0-10] post-treatment; p = 0.004). Collectively, researchers estimated that this experimental therapy led to a savings of $3.6 million in treatment costs that would have otherwise been incurred with exogenous factor IX concentrate administration.

Image: PD

©2017 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

Tags: gene therapygenetic mutationhemophilia
Previous Post

Pharmacomechanical thrombolysis does not lower risk of post-thrombotic syndrome: The ATTRACT trial

Next Post

Declining blood pressure in elderly associated with subcortical microinfarcts

RelatedReports

Video-based behavioral intervention benefits clinical skin examinations
Chronic Disease

Beremagene geperpavec improves wound healing in dystrophic epidermolysis bullosa

December 27, 2022
Separate panels of microRNAs may aid in early detection of pancreatic cancer
Chronic Disease

Low-dose AAVS3 gene therapy is efficacious for treating patients with hemophilia B

July 25, 2022
New genetic link in pulmonary arterial hypertension holds therapeutic promise
Chronic Disease

Valoctocogene roxaparvovec gene therapy efficacious for hemophilia A treatment

March 21, 2022
Blood type correlates with egg reserve in infertile women
Chronic Disease

Betibeglogene autotemcel gene therapy effective for β-thalassemia

February 7, 2022
Next Post

Declining blood pressure in elderly associated with subcortical microinfarcts

Pediatric DKA associated with recent acute care visits

Individualized glycemic control for type 2 diabetes may save the U.S. billions annually

No long-term outcome differences between stenting versus surgery for carotid stenosis

Use of carotid artery stenting increased amongst older patients in the US despite results from CREST trial

License Our Award-Winning Physician-Written Medical News and Visual Abstracts

2 Minute Medicine is the leading authoritative medical news licensing service, and the only with reports written by practicing doctors.

LICENSE CONTENT

2MM+ Premium Access

No ads & unlimited access to all current reports, over 9000 searchable archived reports, visual abstracts, Weekly Rewinds, and the online edition of The Classics Series™ textbook.

Subscription Options
2 Minute Medicine

2 Minute Medicine® is an award winning, physician-run, expert medical media company. Our content is curated, written and edited by practicing health professionals who have clinical and scientific expertise in their field of reporting. Our editorial management team is comprised of highly-trained MD physicians. Join numerous brands, companies, and hospitals who trust our licensed content.

Recent Reports

  • BNT162b2 booster is safe and reduces COVID-19 transmission in older adults
  • Bisphosphonates, denosumab, abaloparatide, teriparatide, and romosozumab reduce postmenopausal fracture risk
  • Epstein-Barr viral load monitoring reduces risk of post-liver transplant lymphoproliferative disease
License Content
Terms of Use | Disclaimer
Cookie Policy
Privacy Statement (EU)
Disclaimer

© 2021 2 Minute Medicine, Inc. - Physician-written medical news.

  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • Account
    • Subscribe
    • Sign-in
    • My account

© 2021 2 Minute Medicine, Inc. - Physician-written medical news.

Want more physician-written
medical news?

Join over 10 million yearly readers and numerous companies. For healthcare professionals
and the public.

Subscribe for free today!

Subscription options