2025 Grant:  Delphi Global Consensus on Clinical Mangement of WAS

Delphi Consensus on Clinical Management of WAS has been launched

We are delighted to share the launch of the Delphi Consensus on the Clinical Management of Wiskott-Aldrich Syndrome - a historic, first-of its kind global effort to create clear, expert-driven guidelines for the care of individuals living with WAS. This initiative brings together the voices of clinicians, mental health experts, patients, caregivers and advocates-building a truly global standard of care that will improve the lives of patients and families with WAS, no matter where they live.  This consensus was made possible through an unrestricted grant from the Wiskott-Aldrich Foundation to Springer Health + (Italy), who are providing the guidance, logistical and methodological support through the Delphi Process.  We are grateful to the Israeli WAS Association for their collaboration and support. 

 Why is this consensus important?  This consensus represents a critical advancement for the Wiskott-Aldrich Syndrome (WAS) community. At present, patients, families, and clinicians often face complex decisions without clear, evidence-based guidance—particularly in cases involving milder forms of WAS or symptomatic carriers. Furthermore, there are currently no standardized recommendations addressing the psychosocial needs of patients, caregivers, and siblings. As gene therapy emerges as a promising treatment option, a lack of consensus also remains regarding patient selection and optimal use. This initiative seeks to address these gaps and establish a unified, expert-driven framework for care.

Objective of the Consensus: To systematically gather and refine expert opinions across key domains, including classification and scoring, indications for hematopoietic stem cell transplantation (HSCT) and gene therapy (GT), management of non-transplanted patients, psychosocial and mental health considerations, and care of female carriers.

What this means for our patients and families:

• Updated guidance on classification of WAS

• Updated management of severe and less severe forms of WAS

• Recommendations on when and for whom HSCT (transplant) or gene therapy may be appropriate

• Best practices for non-transplanted patients

• Guidance on mental health and psychosocial support for patients and families

• Management guidance for female carriers of WAS

Delphi Methodology and Expert Panel Member Involvement: The project will use the Delphi method, a structured and iterative approach to collect opinions from experts to reach consensus. The Scientific Advisory Committee (Drs. Alessandro Aiuti, Michael Albert, Siobhan Burns, Fabio Candotti, Sumathi Iyengar, Christina Mangurian, Luigi Notarangelo and Sung Yun Pai) frame the statements on WAS where consensus ins needed.  This survey is sent out to the WAS Expert Panel to answer in an anonymous survey.  The methodology will include three survey rounds. In between each round, the Scientific Committee analyzes the results, and based on the findings, refine the statements to be submitted to the panel in a second and third round. The results are then finalized and submitted for publication.

Who are the key players:

• 8 Key Opinion Leaders forming the Scientific Advisory Committee

• Methodological support by Springer Health

• 59 WAS experts from around the world

• 15 mental health professionals

• The Israeli WAS Association

When will this be made available? We anticipate publication in Spring of 2026 when all three rounds of Delphi voting will be completed.

From all of us in the WAS community, a heartfelt THANK YOU to our donors and supporters. Your generosity makes life-changing progress possible.

.