Ruxolitinib may be a promising second-line treatment option for pediatric patients with steroid refractory advanced graft-versus-host disease (aGVHD), according to a new study. Based on the findings, researchers suggest their results be validated in a prospective large-scale pharmacokinetic and efficacy trial.
The small retrospective study of 29 patients recruited from 15 pediatric transplantation centers demonstrated that after 28 days, 6 patients achieved a complete response (CR). By 41 days, 19 (65.5%) patients achieved a CR. Another 2 patients had a partial response (PR). Among the 16 patients who were under the age of 6 years, there was a 75% CR and 6.3% PR. All patients who achieved a response discontinued corticosteroid treatment.
Prior to ruxolitinib, patients received an average of 2 immunosuppressive agents, with a range of 1-6.
The prognosis of patients with aGVHD depends mainly on the response of steroid treatment, which is known to be effective in approximately 50% of cases. Indeed, the mortality rate of steroid-refractory or recurrent aGVHD remains as high as 50% to 70%, wrote the researchers. There is currently no standardized second-line strategy for steroid resistant aGVHD.
While several immunosuppressive therapies are used in the second line of treatment, they carry variable response rates. For example, Monoclonal anti-TNF have demonstrated response rates between 30% and 60%. But, relapse at discontinuation was high at approximately 80%.
Results of the current study led the researchers to argue that ruxolitinib may be a possible treatment for these patients, including in cases of gastrointestinal involvement or severe GVHD.
A total of 8 patients experienced treatment failure, and 23 patients were alive after a median follow-up of 685 days after their hematopoietic stem cell transplantation and a median 480 days after initiating ruxolitinib treatment.
CR/PR was a significant factor of survival with a rate of 90% versus 50% in case of treatment failure (P = 0.02), wrote the researchers. Nevertheless, we could not find any association of baseline characteristics and CR/PR to ruxolitinib. Neither the number of immunosuppressive agents before using ruxolitinib nor involved organs was significant.
The researchers were also unable to demonstrate a relationship between ruxolitinib dose and rate of efficacy nor the speed of efficacy. Throughout the study, the median dose of ruxolitinib was 12.6 mg/m2/day.
The study also showed a favorable safety profile of ruxolitinib, with no observed severe hematologic adverse events and with all cytopenias requiring a dose modification resolving.
Viral replication (including cytomegalovirus, Epstein-Barr virus, and adenovirus) was observed in 41.4% of cases indicating that viral replications need to be closely monitored, wrote the researchers. This rate should be interpreted with caution because a comparable frequency of viral replications was reported in children treated with other immunosuppressive drugs, including infliximab, alemtuzumab, and daclizumab.
Laisne L, Neven B, Dalle J, et al. Ruxolitinib in children with steroid-refractory acute graft-versus-host disease: a retrospective multicenter study of the pediatric group of SFGM-TC. Pediatr Blood Cancer. Published online July 2, 2020. doi: 10.1002/pbc.28233.
- Global Animal Stem Cell Therapy Market Research Report by Size, Data, Developments, Global Demand, In-Depth Analysis and Forecast 2020 to 2025 |... - November 27th, 2020
- Stem Cell Therapy Market Size, Growth, Demand, Opportunities & Forecast To 2025 - Cheshire Media - November 27th, 2020
- Celularity Announces Dosing of First Patient in Phase I Study of Human Placental Hematopoietic Stem Cell-Derived Natural Killer Cells (CYNK-001) in... - November 27th, 2020
- Scientists Reveal a New Drug That Directs Stem Cells To Desired Sites - Science Times - November 27th, 2020
- Global Stem Cells Market is estimated to account for US$ 18289.9 Mn by end of 2027, Says Coherent Market Insights (CMI) - Business Wire - November 27th, 2020
- Stem Cell Therapy Market To Observe Exponential Growth By 2020-2027 | Reports Globe - Cheshire Media - November 27th, 2020
- Ash 2020 cell therapy preview the battle for recognition - Vantage - November 27th, 2020
- Animal Stem Cell Therapy Market 2020 to Flourish with an Impressive CAGR of XX% in the year 2026, Market Size & Growth with Complete Business... - November 27th, 2020
- Adipose Tissue-derived Stem Cell Therapy Market: Worldwide Industry to Boost in the Period of 2020-2025 - Express Journal - October 19th, 2020
- Global Stem Cell Therapy Market 2020: Trends, and Opportunity Analysis, Top Manufacturers And Forecast to 2027 - PRnews Leader - October 19th, 2020
- Stem Cell Therapy Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19) - Eurowire - October 19th, 2020
- Global Adipose Tissue-derived Stem Cell Therapy Market 2020 Demand, Business Growing Strategies, Industry Segmentation and Forecast 2025 -... - October 19th, 2020
- Stem Cell Therapy Market Trends, Opportunities, New Technologies and Developments, Competitor analysis and Market Sizing from 2020 to 2026 - PRnews... - October 19th, 2020
- Global Adipose Derived Stem Cell Therapy Market 2020 Demand, Business Growing Strategies, Industry Segmentation and Forecast 2025 - TechnoWeekly - October 19th, 2020
- Notch Therapeutics Strengthens Leadership Team with Appointment of Kamran Alam as CFO and Gregory Block as SVP, Corporate Development - PRNewswire - October 19th, 2020
- Stem Cell Therapy Market To Observe Exponential Growth By 2020-2027 | Verified Market Research - Stock Market Vista - October 19th, 2020
- Stem Cell Therapy Market 2020 growth factors, latest trend and regional analysis of leading players by 2025 - News by Decresearch - October 19th, 2020
- Cell Therapy Market Size, Share, Market Research and Industry Forecast Report, 2020-2027 (Includes Business Impact of COVID-19) - Eurowire - October 19th, 2020
- Autologous Cell Therapy Market is Anticipated to Expand at a CAGR of 18.1% from 2019 to 2027 - Eurowire - October 19th, 2020
- Cell Therapy Market Trend,COVID-19 Impact,Current Industry Figures With Demand By Countries And Future Growth - PRnews Leader - October 19th, 2020
- Gamida Cell and Be The Match BioTherapies Expand Strategic Collaboration - BioSpace - October 19th, 2020
- Cell Therapy Manufacturing Market to be Worth USD 8 billion by 2030, predicts Roots Analysis - The Think Curiouser - October 19th, 2020
- Novellus Therapeutics Exclusively Licenses Induced Mesenchymal Stem Cells (iMSCs) to NoveCite for COVID-19 Related Acute Respiratory Distress Syndrome... - October 19th, 2020
- UofL-born company secures an additional $115 million to advance cell therapy - uoflnews.com - October 19th, 2020
- Global and United States Cancer Stem Cell Therapy Market Leading Players Expected to Witness the Highest Growth 2025 | AVIVA BioSciences, AdnaGen,... - October 19th, 2020
- Can Stem Cell Therapy Repair Damaged Knees? - September 2nd, 2020
- Vor Biopharma and Metagenomi to Collaborate on Engineered Hematopoietic Stem-Cell Therapies - Business Wire - September 2nd, 2020
- COVID-19 cell therapy drives Mesoblast to seek manufacturing muscle - BioPharma-Reporter.com - September 2nd, 2020
- New Report Begins a New Era of Stem Cell Science and Medicine: Stem Cell Biotechnology Company Asymmetrex Tells How It Counts Therapeutic Tissue Stem... - September 2nd, 2020
- Optimized Freezing Solutions for Clinical Application of Cell Therapy Products - Technology Networks - September 2nd, 2020
- Global Cord Blood Banking Market 2020 with Analysis of 44 Industry Players - PRNewswire - September 2nd, 2020
- Cartesian Therapeutics Initiates Clinical Trial of First RNA-Engineered Cell Therapy for Acute Respiratory Distress Syndrome and COVID-19 - PRNewswire - September 2nd, 2020
- New Comprehensive Report on Stem Cell Therapy Market to Witness an Outstanding Growth during 2020 2025 with Top Players Like Chiesi Pharmaceuticals,... - September 2nd, 2020
- CAR T-Cell Optimization Starts in Production, Extends to Therapy - Genetic Engineering & Biotechnology News - September 2nd, 2020
- Growing Focus on R&D Likely to Accelerate the Growth of the Stem Cell Therapy Market - The News Brok - September 2nd, 2020
- Oldest male polar bear on record humanely euthanized at Point Defiance Zoo - MyNorthwest.com - September 2nd, 2020
- FDA Approves First Maintenance Therapy for AML - Medscape - September 2nd, 2020
- Elixirgen Therapeutics planning to begin Phase I/II Clinical Trials of its COVID-19 Vaccine Candidate EXG-5003 at Fujita Health University -... - September 2nd, 2020
- Hill Highlights the Potential of Selinexor as a Less Intensive Option for DLBCL - OncLive - September 2nd, 2020
- Global Rheumatoid Arthritis Stem Cell Therapy Market Dynamics, Forecast, Analysis and Supply Demand 2018 to 2028 - Scientect - September 2nd, 2020
- Growing at an annualized rate of over 20%, the cell therapy manufacturing market is estimated to reach close to USD 10 Billion by 2030, claims Roots... - September 2nd, 2020
- Strategic Analysis to Understand the Competitive Outlook of Cell Therapy Manufacturing Market - The News Brok - September 2nd, 2020
- In Depth Analysis and Survey of COVID-19 Pandemic Impact on Global Canine Stem Cell Therapy Coronavirus Impact Editon of Key Players VETSTEM... - August 10th, 2020
- Tevogen Bio Announces Partnership With Preeminent Scientist Professor Neal Flomenberg, MD, to Investigate Proprietary T-Cell Therapy for Treatment of... - August 10th, 2020
- Marker Therapeutics Reports Second Quarter 2020 Operating and Financial Results - PRNewswire - August 10th, 2020
- University of Minnesota expands clinical investigation of engineered iPSC-derived natural killer cells, opening U.S. clinical trial for the treatment... - August 10th, 2020
- Clinical study using mesenchymal stem cells for the treatment of patients with severe COVID-19 - DocWire News - August 10th, 2020
- UPDATED: After a 4-year sojourn, struggling microbiome pioneer Seres claims a breakout PhIII comeback. And shares respond in frenzied spike -... - August 10th, 2020
- New Report: Regenerative Medicine & Advanced Therapies Sector Thriving Despite COVID-19 - PharmiWeb.com - August 10th, 2020
- Covid-19 roundup: CureVac beefs up its unicorn IPO dreams as billionaire owner takes this Covid-19 mRNA player on a forced march to Nasdaq; Kodak's... - August 10th, 2020
- Coronavirus Drug and Treatment Tracker - The New York Times - August 10th, 2020
- Nearly 70K donated so far towards Wistaston boy's 120K lifesaving treatment - Cheshire Live - August 10th, 2020
- Avacta extends partnership with Daewoong Pharmaceutical and AffyXell Therapeutics for Covid-19 stem cell treatments - Cambridge Independent - August 9th, 2020
- Three Years After Stem Cell Trial for Heart Failure was Abandoned New Evidence Shows it Works - Diagnostic and Interventional Cardiology - August 9th, 2020
- Stem Cell And Platelet Rich Plasma (PRP) Alopecia Therapies Market 2020 Size by Product Analysis, Application, End-Users, Regional Outlook,... - August 9th, 2020
- IMAC Holdings Receives FDA Authorization to Initiate Clinical Study of Its Umbilical Cord-Derived Allogenic Mesenchymal Stem Cells for the Treatment... - August 9th, 2020
- Strathmore man with MS calls for more Canadian treatment alternatives - CTV Toronto - August 9th, 2020
- Wave of New Therapies Improve Outcomes for Patients with Multiple Myeloma - Dana-Farber Cancer Institute - August 9th, 2020
- Adipose Derived Stem Cell Therapy Market: Technological Advancement & Growth Analysis with Forecast to 2026 - Chelanpress - August 9th, 2020
- ASCO Expert on the Possible Benchmark Analysis of Autologous Transplantation in B-Cell Lymphomas - Cancer Network - August 9th, 2020
- Cell Therapy Market: Study Navigating the Future Growth Outlook | Osiris Therapeutics, NuVasive, Vericel Corporation - Chelanpress - August 9th, 2020
- Global Stem Cell Therapy Market 2020: Growth, Demand, Service, Types, Applications, Key Players and Industry Forecast till 2025 - Chelanpress - August 9th, 2020
- Personalized Cell Therapies Market to Show Incredible Growth by 2025 - Owned - August 9th, 2020
- Nerve Repair and Regeneration Market worth $9.7 billion by 2025 - Exclusive Report by MarketsandMarkets - PRNewswire - August 9th, 2020
- High-throughput 3D screening for differentiation of hPSC-derived cell therapy candidates - Science Advances - August 9th, 2020
- Immatics Extends Cell Therapy Manufacturing Collaboration with UTHealth - GlobeNewswire - August 9th, 2020
- Contract Manufacturing Services market for stem cells is expected to be worth over USD 2.5 billion by 2030, claims Roots Analysis - Market Research... - August 9th, 2020
- In Depth Analysis and Survey of COVID-19 Pandemic Impact on Global Cancer Stem Cell Market Report 2020 Key Players Thermo Fisher Scientific Inc.,... - August 9th, 2020
- Tafasitamab Combination Approved for Adults With R/R DLBCL - AJMC.com Managed Markets Network - August 8th, 2020
- Celularity announces the activation of first California Clinical Trial Site following CIRM Grant Award to Advance Treatments for COVID-19 - PRNewswire - August 8th, 2020
- Trending News on Targeted Oncology, Week of August 7, 2020 - Targeted Oncology - August 8th, 2020
- Find Out Why Bioreactor Market is thriving worldwide by top key players like mergency Response USA, Buddi US, LLC, Connect America - Owned - August 8th, 2020
- BCLI: Phase 3 ALS Data Expected by the End of November 2020 - Zacks Small Cap Research - August 8th, 2020
- New Report: Regenerative Medicine & Advanced Therapies Sector Thriving Despite COVID-19 - GlobeNewswire - August 8th, 2020
- Immatics Extends Cell Therapy Manufacturing Collaboration with UTHealth - marketscreener.com - August 8th, 2020
- Impact of COVID-19 Outbreak on Canine Stem Cell Therapy Market Expected to Secure Notable Revenue Share During 2020-2026 - Research Newspaper - August 8th, 2020
- Calidi Biotherapeutics in San Diego receives FDA Approval for COVID-19 Treatment clinical trial - - KUSI - August 7th, 2020
- Edited Transcript of BLCM.OQ earnings conference call or presentation 6-Aug-20 9:00pm GMT - Yahoo Finance - August 7th, 2020
- NICE Amends Guidance on Haematopoietic Stem Cell Transplantation During the Pandemic - Medscape - August 6th, 2020
- AlloVir raises $276M IPO to run broad cell therapy program - FierceBiotech - August 6th, 2020