Healthcare Professionals

This page is intended for healthcare professionals.

GvHD: Pathology & Epidemiology

Gut Microbiome & Blood Cancers

Early Access Program

Clinical Trials, Scientific Publications

GvHD : Pathology & Epidemiology

Graft-versus-Host Disease (GvHD) is one of the potential complications following an allogeneic hematopoietic stem cell transplantation (HSCT). It is estimated that 30 to 50% of patients develop GvHD after an allogeneic HSCT.

There are two types of GvHD, distinguished notably by the timing of symptom onset: symptoms occurring within 100 days after transplantation are classified as acute GvHD, while those appearing later are considered chronic GvHD. The symptoms also differ between these two forms.

  • Chronic GvHD presents with more diffuse symptoms. It can affect the skin, eyes, mouth, muscles, and gastrointestinal tract, and may lead to infections and breathing difficulties.
  • Acute GvHD, on the other hand, primarily targets three organs: skin, liver, and gastrointestinal tract. Acute GvHD can be severe and rapidly fatal. It is one of the most feared complications following allogeneic HSCT and generally has a poorer prognosis.

During an allo-HSCT, despite careful donor selection based on HLA matching, the graft can attack the patient’s tissues, recognizing them as foreign.

The severity of GvHD is graded on a scale from 0 (no reaction) to 4 (severe reaction):

The Gut Microbiome: An Innovative Approach in the Management of Blood Cancers

In hematologic malignancies, the gut microbiome plays a crucial role in modulating immune response and enhancing treatment efficacy. Dysbiosis caused by cancer itself, chemotherapy, or antibiotics may trigger systemic inflammation and impact patient outcomes. Emerging evidence shows that microbiota restoration can improve treatment tolerance, lower the risk of complications such as graft-versus-host disease (GvHD) following allogeneic stem cell transplantation, and potentially extend overall survival. These advances pave the way for novel microbiome-based therapeutic strategies in hemato-oncology.

Read more 👇

Early Access Program

Clinical trials are essential to demonstrate the safety and efficacy of new treatments, enabling regulatory authorities to approve their market authorization. However, when no therapeutic alternatives exist for patients with significant medical needs, specific mechanisms allow early access to medicines prior to official approval, under strict conditions and with the agreement of competent authorities. The Compassionate Use Authorization (also called Early Access Program), granted by the French National Agency for Medicines and Health Products Safety (ANSM) or other national authorities, permits the exceptional use of a drug candidate for a specific indication either in the absence of a marketing authorization application or before such authorization is granted.

To submit an EAP request, all the following criteria must be met:

  • The medicine is intended to treat a serious, rare, or debilitating disease,
  • No appropriate treatment is available,
  • The medicine is not involved in a clinical trial with human subjects (CTHS) for commercial purposes,
  • Or, if the medicine is part of a commercial clinical trial, the patient cannot participate, treatment cannot be delayed, and the company has committed to submitting an early access request,
  • The medicine’s efficacy and safety are presumed based on available clinical data.

MaaT Pharma works closely with the ANSM to monitor patients receiving compassionate access according to the Therapeutic Use and Patient Monitoring Protocol (PUT-SP), when applicable. The list of medicinal products available under compassionate use is available on the ANSM website. All requests for compassionate use access must be submitted via the ANSM’s “e-Saturne” platform.

For any questions regarding the compassionate use program, please contact us.

Clinical trials, clinical data, and scientific publications

Clinical Trials

Clinical Data

Topline Results for Phase III (MaaT013): ARES

Early Access Program

Scientific Publications

Managing GvHD

Gut Microbiome & GvHD

Manufacturing & Processes