Expectations and experiences: Parent and patient perspectives

Recently published in the August 2021 Issue of Clinical Immunology is an article entitled "Expectations and experience: Parent and patient perspectives regarding treatment for Severe Combined Immunodeficiency (SCID)", co-authored by Heather Smith, Christopher Scalchunes, Morton J.Cowan, JenniferPuck, and JenniferHeimall. The survey was designed and distributed to families of individuals with SCID. Among the questions were SCID genotype and treatment, individual recollections of pre-treatment counseling, and present clinical status.

Of the 151 surveys analyzed:

• 132 treated with hematopoietic cell transplantation (HCT), 19 with gene therapy (GT)
• 37% expected that their treatment would lead to a "cure"
• 43% expected the treatment to last a lifetime
• of 136 living patients, 59% reported good health overall; 65% reported some persistent health challenges

The key finding of the study included differing expectations of long-term outcomes following treatment than the intended provider communication. Healthcare providers must emphasize the possible need for ongoing treatments following HCT for SCID.  In addition, counseling is important throughout each phase of the treatment journey. Lifelong medical follow-up is critical for patients with SCID in order to recognize and manage post-treatment adverse outcomes. 

For some, interpretation of the word “cure” varied, leading to misunderstanding regarding the need for continued medical evaluations and additional therapies. Clear communication regarding the importance of lifelong follow-up, no matter the treatment outcome, will help to optimize good health and quality of life.

CLICK HERE>> to read the full article.