Archive for category: News

Researchers have found in a new research that previous gastrectomy was as an independent risk factor for post-operative bile leakage (POBL) in patients undergoing laparoscopic liver resection (LLR). Further in order to reduce the incidence of POBL in high-risk cases, meticulous surgical techniques and comprehensive perioperative management are essential.

The American College of Chest Physicians® (CHEST) recently released a new clinical guideline on the transfusion of fresh frozen plasma (FFP) andplatelets in critically ill adults. Published in the journal CHEST®, the guideline contains seven evidence-based recommendations to provide a framework for clinicians to implement in their own facilities.

“In practice, we see all too often [that] prophylactic transfusion of platelets or fresh frozen plasma is done unnecessarily,” says Angel Coz Yataco, MD, FCCP, lead author on the guideline. “Transfusion is rarely needed prior to bedside procedures, with lumbar puncture being a possible exception. Following the new guideline, we can drastically reduce the use of FFP.”

The panel of experts developed seven Population, Intervention, Comparator, and Outcome questions addressing platelet and FFP transfusions in critically ill patients and performed a comprehensive evidence review. The panel applied the Grading of Recommendations, Assessment, Development, and Evaluations approach to assess the certainty of evidence and to formulate and grade recommendations.

“In the United States, 20% of platelets and FFP are transfused to critically ill patients, totaling 2.2 million units of each annually,” says Dr. Coz Yataco. “They are scarce resources with variable costs and access globally. If implemented on a large scale, this guideline provides the framework to decrease the use [to] approximately half a million less units of platelets and FFP transfused annually in the United States alone.”

The panel formulated seven conditional recommendations. In addition to four procedure-specific recommendations, the guideline recommends:

• In stable non-bleeding critically ill patients with thrombocytopenia and without high risk of spontaneous bleeding, we suggest transfusing platelets if platelet counts fall below 10 × 109/L (Conditional recommendation, very low certainty of evidence).

• In stable non-bleeding critically ill patients with thrombocytopenia who are considered at high risk of spontaneous bleeding, we suggest transfusing platelets if platelet counts fall below 30-50 × 109/L (Conditional recommendation, very low certainty of evidence).

• In critically ill patients with thrombocytopenia and serious active bleeding, we suggest transfusing platelets if platelet counts fall below 50 × 109/L (Conditional recommendation, very low certainty of evidence).

The entire list of recommendations included in the new guideline are as follows-

Summary of Recommendations

The recommendations in this document apply to critically ill patients, excluding trauma and neuro-critical care populations. These recommendations should be implemented in a hierarchal fashion. Recommendations 1-3 should be applied first to critically ill patients with thrombocytopenia, while recommendations 4-7 provide additional guidance for specific situations involving invasive procedures.

1. In stable non-bleeding critically ill patients with thrombocytopenia and without high risk of spontaneous bleeding, we suggest transfusing platelets if platelet counts fall below 10 × 109/L (Conditional recommendation, very low certainty of evidence).

2. In stable non-bleeding critically ill patients with thrombocytopenia who are considered at high risk of spontaneous bleeding, we suggest transfusing platelets if platelet counts fall below 30-50 × 109/L (Conditional recommendation, very low certainty of evidence).

3. In critically ill patients with thrombocytopenia and serious active bleeding, we suggest transfusing platelets if platelet counts fall below 50 × 109/L (Conditional recommendation, very low certainty of evidence)

4. Vascular procedures

4 A. In critically ill patients at increased risk of bleeding due to thrombocytopenia undergoing a central venous catheter or arterial line insertion, we suggest against routine prophylactic platelet transfusion (Conditional recommendation, very low certainty of evidence).

4 B. In critically ill patients at increased risk of bleeding due to coagulopathy undergoing a central venous catheter or arterial line insertion, we suggest against routine prophylactic FFP transfusion (Conditional recommendation, very low certainty of evidence).

5. Bedside thoracic or abdominal procedure

5 A. In critically ill patients with increased risk of bleeding due to thrombocytopenia undergoing a bedside thoracentesis or paracentesis, we suggest against routine prophylactic platelet transfusion (Conditional recommendation, very low certainty of evidence).

5 B. In critically ill patients with increased risk of bleeding due to coagulopathy undergoing a bedside thoracentesis or paracentesis, we suggest against routine prophylactic FFP transfusion (Conditional recommendation, very low certainty of evidence).

6. Lumbar Puncture

6 A. In critically ill patients with increased risk of bleeding due to thrombocytopenia undergoing a bedside lumbar puncture, we suggest platelet transfusion if platelets counts are 40-50 × 109/L or lower (Conditional recommendation, very low certainty of evidence).

6 B. In critically ill patients with increased risk of bleeding due to coagulopathy undergoing a bedside lumbar puncture, we suggest FFP transfusion to target INR 1.5-2 before the procedure (Conditional recommendation, very low certainty of evidence).

7. Bedside Endoscopy

Bronchoscopy

7 A. In critically ill patients with increased risk of bleeding due to thrombocytopenia undergoing routine flexible bronchoscopy without biopsy, we suggest against routine prophylactic platelet transfusion (Conditional recommendation, very low certainty of evidence).

7 B. In critically ill patients with increased risk of bleeding due to coagulopathy undergoing a routine bedside flexible bronchoscopy without biopsy, we suggest against routine prophylactic FFP transfusion (Conditional recommendation, very low certainty of evidence).

GI Endoscopy

7 C. In critically ill patients with suspected portal hypertension related GI bleeding and increased risk of bleeding due to thrombocytopenia who are undergoing GI-endoscopy, we suggest against routine platelet transfusion (Conditional recommendation, very low certainty of evidence)

7 D. In critically ill patients with suspected portal hypertension related bleeding and increased risk of bleeding due to coagulopathy who are undergoing GI-endoscopy, we suggest against routine FFP transfusion (Conditional recommendation, very low certainty of evidence).

Reference:

Yataco, Angel Coz et al., Transfusion of Fresh Frozen Plasma and Platelets in Critically Ill Adults, CHEST Journal, DOI:10.1016/j.chest.2025.02.029 

The U.S. Food and Drug Administration (FDA) has approved prademagene zamikeracel (Zevaskyn) for the treatment of blistering wounds caused by recessive dystrophic epidermolysis bullosa (RDEB) in both adults and children, according to an announcement by Abeona Therapeutics.

There is no cure for RDEB and ZEVASKYN is the only FDA-approved product to treat RDEB wounds with a single application.

“Today’s approval of ZEVASKYN represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease,” said Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona. “We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, ZEVASKYN can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase 3 study.”

First-of-its-kind gene therapy with robust body of clinical evidence

The FDA approval of ZEVASKYN is based on the pivotal Phase 3 VIITAL™ study (NCT04227106), a multi-center, randomized, intrapatient-controlled trial that met its two co-primary efficacy endpoints demonstrating statistically significant healing of 50 percent or more from baseline in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at six months after treatment.

Across 43 large and chronic wounds treated with a single application of ZEVASKYN, 81 percent of wounds showed 50 percent or more healing (P<0.0001) as evaluated at six months, compared to 16 percent in 43 matched control wounds treated with standard of care. The most common adverse events were observed in fewer than five percent of patients and included procedural pain and itch.

“ZEVASKYN was well-tolerated and efficacious in clinical studies, providing clinically meaningful improvements in wound healing, pain reduction, and other associated symptoms in large chronic RDEB wounds after a single application,” said Jean Tang, M.D., Ph.D., professor of dermatology and lead principal investigator of the VIITAL™ study. “In the completed Phase 1/2a study of ZEVASKYN, we have observed wound healing and pain reduction that have lasted for years after a single application. Today we can celebrate the availability of an exciting new therapeutic option made possible by the incredible courage of patients and families who participated in these clinical studies.”

In the Phase 1/2a study of ZEVASKYN (NCT01263379), a single center, open label study in 38 chronic wounds across 7 patients showed that a single surgical application of ZEVASKYN was associated with long-term improvement at treated sites over a median follow-up of 6.9 years; range 4 to 8 years.

“After many years of work, it is great to see this FDA approval of ZEVASKYN. The EB patients deserve all that we can do for them,” said M. Peter Marinkovich, M.D., associate professor of dermatology and co-principal investigator of the VIITAL™ study.

“Based on the strength of our data across clinical trials, we are confident in ZEVASKYN’s ability to deliver long-term results after a single treatment application,” said Madhav Vasanthavada, Ph.D., M.B.A., Chief Commercial Officer of Abeona. “We are committed to working closely with both commercial and government payers on outcome-based agreements that stand behind the promise of ZEVASKYN for patients, and expedite access.”

Across both clinical studies, ZEVASKYN was well-tolerated with no treatment-related serious adverse events observed to date.

Anna L. Bruckner, MD, Co-Director of the EB Clinic at Children’s Hospital of Colorado and Professor of Dermatology, University of Colorado School of Medicine, said, “The FDA approval of ZEVASKYN marks a monumental step forward for individuals living with RDEB and their families, offering a much-needed, long-lasting treatment option for this devastating condition and providing hope for improved quality of life for these patients.”

Amy Paller, M.D., pediatric dermatologist and clinical researcher, said, “Grafting gene-corrected skin onto chronically open wounds of patients with recessive dystrophic epidermolysis bullosa promises the potential to provide long-term healing of wounds, reduction in pain and reduced risk of infection. This therapeutic option will nicely complement recently approved topical products.”

Dr. Seshadri added, “We are grateful to the patients, their families, and caregivers for their support of ZEVASKYN. We express our gratitude to debra of America for their unwavering support throughout the development journey, in particular, for their interactions with the FDA in support of ZEVASKYN and on behalf of the EB community that have helped make today’s regulatory approval possible. We are also thankful for the clinical study investigators, study site personnel, and the entire Abeona team for their collective commitment and determination through the development process, and contribution to this milestone achievement. We look forward to providing the RDEB community access to now-approved ZEVASKYN.”

Addressing the underlying cause of RDEB

With mutations in both copies of the COL7A1 gene that expresses Type VII collagen, people with RDEB have extremely fragile skin characterized by extensive blistering and severe wounds that often cover more than 30 percent of a patient’s body surface, and in some cases up to 80 percent. RDEB wounds cause debilitating pain and systemic complications impacting the length and quality of life. These wounds are difficult to heal, can remain open for years, and many that do close tend to reopen.

ZEVASKYN consists of a patient’s own skin cells (keratinocytes) that have been genetically modified, to produce functional Type VII collagen. ZEVASKYN sheets are surgically applied to the patient’s wounded areas. In a single application of ZEVASKYN, up to 12 credit card-sized sheets can be joined together to cover large areas or applied to multiple distinct wounds, allowing for signficant coverage of affected body areas.

Brett Kopelan, M.A., Executive Director of debra of America, the only national advocacy organization that provides all-inclusive care to the epidermolysis bullosa community, and father to Rafi, a teenager with RDEB, said, “I, and the team at debra of America, are very excited about the FDA’s approval of ZEVASKYN. Given that this therapeutic product addresses even the largest, most difficult, and problematic chronic wounds, we believe that the application of ZEVASKYN can significantly increase the quality of life of patients. Furthermore, I believe ZEVASKYN has the potential to transform the day-to-day standard of care for patients who suffer with these large chronic nonhealing wounds that cause significant pain and stress not only for the patient, but also for their caregivers. We are honored to have formed such a close relationship with Abeona over the years and look forward to deepening our partnership by helping ensure there is broad access for the patient population to ZEVASKYN, which I know is their ultimate goal.”

Michael Hund, M.B.A., Chief Executive Officer of EB Research Partnership (EBRP), the largest global organization dedicated to funding research to treat and cure epidermolysis bullosa (EB), said, “The mission of EBRP is to advance commercially sustainable research aimed at treating and ultimately curing EB. We are honored to partner with the entire Abeona team and commend their leadership, determination, and passion to deliver much needed innovative solutions for individuals and their families living with EB. They continue to share our values and commitment to accelerate treatments to the EB community as quickly as possible. Abeona’s development and advancement of ZEVASKYN delivers a landmark moment for the global EB community, and their leadership in gene therapy holds so much promise to innovate the therapeutic landscape for not only EB, but many other rare diseases and conditions. EBRP is looking forward to collaborating with Abeona to continue to support the EB community.”

ZEVASKYN availability in the third quarter of 2025

ZEVASKYN is expected to be available beginning in the third quarter of 2025 through ZEVASKYN Qualified Treatment Centers (QTCs). The QTCs are well-recognized epidermolysis bullosa treatment centers with cell and gene therapy experience, situated across the U.S. to ensure patients nationwide have access to this important treatment.

Abeona’s comprehensive patient support program, Abeona Assist™, offers personalized support, including helping patients understand their insurance benefits and financial assistance options, and providing travel and logistical assistance for eligible patients. 

“We are grateful to the dedicated scientists whose work over the past decade made the development of ZEVASKYN possible,” said Marissa Perman, MD, Section Chief of Dermatology and Director of the Epidermolysis Bullosa Multidisciplinary Clinic at Children’s Hospital of Philadelphia and paid consultant to Abeona. “Having a new, uniquely differentiated, gene therapy for our patients with RDEB is a significant milestone in helping these special patients live fuller, pain-free and itch-free lives with less wounds. As a physician caring for patients with RDEB, I look forward to the opportunity to see this treatment in our practice.”

Joyce Teng, MD, PhD, professor in dermatology with multiple hospital affiliations, said, “I’m thrilled to celebrate a groundbreaking advancement in therapeutic development for recessive dystrophic EB, a condition that has long needed an innovative solution. ZEVASKYN offers additional hope for patients and families affected by this painful and devastating skin disorder. This milestone is a testament to the dedication of scientists, researchers and medical professionals who have worked tirelessly to bring cutting edge treatments to those in need. It represents a scientific triumph, a profound step toward improving quality of life for individuals affected. We look forward to seeing the impact that this therapy will have on so many lives.”

In connection with the FDA approval, Abeona received a Rare Pediatric Disease Priority Review Voucher (PRV). The Company plans to monetize the PRV.

About Recessive Dystrophic Epidermolysis Bullosa

Recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without a cure, is characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in both copies of the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin.

About ZEVASKYN™ (prademagene zamikeracel) gene-modified cellular sheets or pz-cel

ZEVASKYN is the first and only autologous cell sheet-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe skin disease caused by a defect in both copies of the COL7A1 gene resulting in the inability to produce functional type VII collagen. Without functional type VII collagen and anchoring fibrils, the skin is fragile and blisters easily, leading to wounds that continually open and close, or fail to heal altogether. Patients often have large open wounds that are at a high risk of systemic infection. ZEVASKYN incorporates the functional type VII collagen-producing COL7A1 gene into a patient’s own skin cells, ex vivo, using a replication-incompetent retroviral vector to produce functional type VII collagen in treated wounds. ZEVASKYN has demonstrated clinically meaningful wound healing and pain reduction with a single surgical application.

There has been a troubling rise in adolescent mental health struggles and suicide rates over the past decade, with a dramatic increase following the start of the COVID-19 pandemic. This crisis has been accompanied by an increased demand for pediatric inpatient psychiatry units (IPUs) across the United States. However, despite the growing need, which has reached the point of bed shortages, the effectiveness of IPUs on teen mental health outcomes remains understudied.

This study, led by Dr. Patricia Ibeziako from Boston Children’s Hospital, reviewed the electronic medical records and self-report questionnaires of over 200 adolescents (ages 12–17) admitted to the IPU between September 2021 and September 2023. Their study was published in the journal Psychiatric Research and Clinical Practice on April 21, 2025. They found one of the highest lifetime rates of suicide attempts ever reported in adolescents, with more than 75% of teens reporting at least one suicide attempt in their lifetime. Furthermore, nearly 70% of participants reported having suicidal thoughts within two weeks before admission, highlighting the severity of this mental health crisis. Depression was the most common diagnosis, with 93% of adolescents meeting the diagnostic criteria. This was often accompanied by other conditions such as anxiety disorders, attention deficit hyperactivity disorder, and trauma-related disorders.

At the hospital, the teens received daily care from doctors, therapists, nurses, and counsellors. They also had access to group therapy, school support, and help with any physical health problems. The team used special tools to assess suicide risk and conduct safety planning. They also used proven therapies like Cognitive Behavioural Therapy (CBT) and Dialectical Behaviour Therapy (DBT), which are known to help people with depression and suicidal thoughts.

Most importantly, the study focused on the teens’ own voices by using Patient-Reported Outcomes (PROs)—surveys that asked the teens how they were feeling and functioning. This helped the care team understand what was working and what needed more attention.

But despite the high severity of symptoms upon admission, the study found that the patients showed significant improvement by the time they were discharged. “Self-reported measures on depression, anxiety, emotional regulation, family functioning, and overall life satisfaction all improved significantly”, says Dr. Ibeziako. “In fact, the largest treatment effect was seen in depressive symptoms, and improvements were observed across all subscales of depression, including mood, suicidal ideation, and energy levels.”

Perhaps most strikingly, the study found that adolescent scores for suicidal thoughts decreased by more than half. “The implementation of enhanced suicide screening and treatment that aligns with the Zero Suicide framework has made a meaningful difference in these young people’s lives,” adds Dr. Ibeziako. This framework, which was introduced during the first year of the pandemic, takes a comprehensive, system-wide approach to suicide prevention. The findings showed that when paired with other treatments for depression, the intervention led to significant improvements in symptoms and emotional regulation. “Pediatric IPUs play a crucial role in delivering these life-saving interventions,” emphasizes Dr. Ibeziako.

The results of this study show that timely and targeted psychiatric care can be lifesaving. With adolescent depression and suicidal thoughts at an all-time high, these results are a clear call to action for healthcare systems to prioritize resources and access to pediatric IPUs and further research into their outcomes.

Reference:

Patricia Ibeziako, Inpatient Psychiatry Patient Reported Outcomes: Adolescent Emotional Distress, Suicidal Thoughts and Behaviors, Psychiatric Research and Clinical Practice, https://doi.org/10.1176/appi.prcp.20240127.