HHT/Hereditary Hemorrhagic Telangiectasia: Hemostatic Agent 'SurgeCell' Gains Additional Indication Approval for Severe Epistaxis — A Major Step Forward Driven by 20,000+ Signatures and Patient-Led Advocacy

Osler's disease / HHT (Hereditary Hemorrhagic Telangiectasia) is a designated intractable disease affecting all medical specialties, characterized by systemic vascular abnormalities. Epistaxis (nosebleeds) is one of its hallmark symptoms. However, nosebleeds in HHT patients differ significantly from common epistaxis. They originate from fragile, multiple telangiectasias and vascular malformations in the nasal cavity. Standard hemostatic procedures such as electrocautery, compression, gauze packing, or laser ablation may temporarily stop bleeding but can subsequently worsen hemorrhage.

The additional indication was approved on May 13, representing a major advancement for patients suffering from recurrent, severe nosebleeds, safeguarding both lives and quality of life.

Regarding official announcement, we have been informed that it will be made simultaneously once all necessary conditions are met.

Background

From April 2024 onward, a critical situation arose where essential hemostatic materials became inaccessible. SurgeCell had been a vital hemostatic agent used by HHT patients under physician guidance to manage severe epistaxis at home.

However, starting April 2024, the previously prescribed hemostatic material was reclassified as a 'surgical material,' making it difficult for patients to receive prescriptions as before.

As a result, patients who had previously managed bleeding at home or in outpatient settings faced increasing difficulty in achieving hemostasis, leading to a rise in emergency requests. Furthermore, even when emergency assistance was sought, patients often could not find accepting facilities, failed to receive appropriate hemostatic treatment for HHT, or required blood transfusions—placing them in life-threatening conditions. (Osler Disease (HHT) Patient Association | Consultation and Information on Recurrent Nosebleeds and Designated Intractable Disease No. 227)

For HHT patients, being denied medical care or facing treatment difficulties due to nosebleeds is not uncommon. The patient association has received numerous inquiries on this issue over many years.

Patient-Led Research and Development of Hemostasis Care

The president of the Japan Osler Disease Patient Association personally researched effective hemostasis methods for HHT-related epistaxis after enduring years of suffering. With the cooperation of a small number of understanding physicians, they conducted extensive research on the appropriate use of SurgeCell for HHT patients.

Appropriate use of SurgeCell has enabled more patients to control their bleeding independently, revealing the potential to avoid emergency transport, incorrect treatments in ENT clinics, blood transfusions due to untreated bleeding, and hospitalization.

However, an abrupt regulatory reclassification left patients unable to access this essential hemostatic material when needed.

Path to Advocacy

The patient association launched a petition campaign demanding stable supply and use of SurgeCell. Supported by widespread endorsements, the association continued its signature campaign, specifically naming HHT-related epistaxis as 'multiple intranasal capillary hemorrhage.'

The association president requested a meeting with the Ministry of Health, Labour and Welfare, submitted formal petitions, and persistently advocated for the realities faced by HHT patients, cases of failed hemostasis, the need to improve incorrect medical practices, and the rationality and cost-effectiveness of self-hemostasis.

Over 20,000 Signatures as the Driving Force!

The petition received support from over 20,000 individuals.

We sincerely thank everyone who signed, those who amplified the voices of patients and families, physicians and healthcare professionals who showed understanding, and representatives from related companies and government agencies.

Patient Association Leadership

This patient-led achievement of additional indication approval is a significant case demonstrating that even for rare and intractable diseases, patient voices, patient association research, collaboration with medical professionals, and sustained advocacy to government bodies can drive systemic change.

Lack of Awareness Among ENT Specialists, Paramedics, and Healthcare Providers Leads to Ongoing Harm

HHT-related epistaxis is not 'ordinary nosebleeds.'

The nature of epistaxis in Osler's disease / HHT differs fundamentally from common nosebleeds.

Bleeding originates from multiple fragile telangiectasias in the nasal cavity. Our association refers to this condition as

'multiple intranasal capillary hemorrhage.'

Standard treatments for common nosebleeds—such as strong compression, electrocautery, laser hemostasis, and gauze packing—may temporarily stop bleeding but are often ineffective in the long term. These methods can damage blood vessels, worsen bleeding, or lead to intractable hemostasis. In the worst cases, nasal septal perforation (a hole forming in the center of the nose) can occur, leading to persistent bleeding difficulties. Several victims have been reported within our association.

Patients, members of the association, have long explained: 'This is not ordinary nosebleeds,' 'pressing hard makes it worse,' and 're-bleeding occurs when removing gauze.' However, due to insufficient understanding in medical and emergency settings, they have endured harsh conditions for years.

Even when information is provided to members, patients report being 'ignored by doctors,' told 'to stop midway,' or 'not to come at night,' leading to severe physical and mental exhaustion. To address this, the association president developed a self-hemostasis protocol using SurgeCell. Over 90% of patients can achieve self-hemostasis with this method, resulting in numerous benefits: improved quality of life, alleviation of severe anemia, reduced psychological burden, fewer emergency transports, reduced burden on physicians, and lower medical costs.

Ongoing Challenges

While the additional indication approval is a major advancement, approval alone does not immediately ensure that all patients nationwide can use SurgeCell with confidence.

Even after approval, numerous procedures remain, including insurance coverage approval by the Central Pharmaceutical Affairs Committee and other administrative processes.

Even with full approval, systemic infrastructure must be established so that all patients can access SurgeCell reliably.

This includes:

- Education for physicians, nurses, pharmacists, and paramedics on HHT-related epistaxis management

- Securing availability of SurgeCell in hospitals and pharmacies

The Japan Osler Disease Patient Association will continue collaborating with the Ministry of Health, Labour and Welfare, related companies, medical institutions, and healthcare professionals to establish a system ensuring that every patient in need can access SurgeCell when necessary.

Message to Healthcare Professionals

Epistaxis in Osler's disease / HHT is pathologically different from common nosebleeds. This type of bleeding is a key sign of Osler's disease.

When patients explain: 'This is different from ordinary nosebleeds,' 'electrocautery, compression, or gauze packing worsens it—please avoid these,' or 'I need to stop bleeding with SurgeCell,' we urge you not to dismiss their experiences and to respond with careful, condition-appropriate care.

For patients with severe recurrent epistaxis, access to appropriate hemostatic materials directly impacts emergency transport, blood transfusions, worsening anemia, sleep disorders, employment difficulties, and the ability to maintain daily life.

Our association will continue to disseminate information to healthcare professionals about the unique nature of HHT-related epistaxis and appropriate hemostatic management.

Comment from the President of the Japan Osler Disease Patient Association

Epistaxis in Osler's disease is not ordinary nosebleeds.

I have presented as a panelist at the 127th Japan Society for Head and Neck Surgery, discussing an overview of Osler's disease and techniques for managing HHT-related epistaxis.

Bleeding originates from 'multiple fragile telangiectasias and vascular malformations' in the nasal cavity. Standard hemostatic procedures can exacerbate the condition, requiring utmost caution beyond life-threatening emergencies.

Patients have endured repeated bleeding episodes, repeatedly explained their condition, yet remained misunderstood—facing delayed emergency admissions, denial of care, and ultimately requiring transfusions or hospitalization.

This additional indication approval for SurgeCell was made possible by the cooperation of over 20,000 signatories, the heartfelt voices of patients and families, and the support of a few understanding physicians.