Google
Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Acceptability of In Utero Hematopoietic Cell Transplantation for Sickle Cell Disease
source: Medical Decision Making
year: 2017
authors: Antiel RM, Halpern SD, Stevens EM, Vrecenak JD, Patterson CA, Tchume-Johnson T, Smith-Whitley K, Peranteau WH, Flake AW, Barakat LP
summary/abstract:BACKGROUND:
In utero hematopoietic cell transplantation (IUHCT) has curative potential for sickle cell disease (SCD) but carries a risk of fetal demise.
METHODS:
We assessed the conditions under which parents of children with SCD and young adults with SCD would consider IUHCT in a future pregnancy, given a 5% fixed risk of fetal demise. Participants were randomized to consider a hypothetical cure rate (20%, 40%, or 70%). Subsequently, cure rate was either increased or decreased depending on the previous answer to reveal the lowest acceptable rate. Participants also completed the Pediatric Research Participation Questionnaire (PRPQ) and an omission scale.
RESULTS:
Overall, 74 of 79 (94%) participants were willing to consider IUHCT, and 52 (66%) participants accepted IUHCT at a cure rate of 40%, the estimated rate of therapeutic mixed chimerism. Participants with higher scores on the PRPQ perceived benefits scale were more likely to participate at lower cure rates (OR 1.08, p=0.007) and participants with a greater degree of omission bias were less likely to participate at lower cure rates (OR 0.83, p=0.04). Demographics and SCD severity were not significantly associated with acceptability of IUHCT.
CONCLUSION:
This study suggests that the majority of parents >and young adults would consider IUHCT under expected therapeutic conditions.
DOI: 10.1177/0272989X17707214
read more full text
+myBinderRelated Content
-
Public Meeting on Patient-Focused Drug Development for Chronic PainMeeting Information: On July 9, 2018, F...
-
Sickle Cell Disease: Managing PainCurrently, sickle cell disease has no wi...
-
Predictive factors of daily opioid use and quality of life in adults with sickle cell diseaseObjectives: In adults with sickle cell ...
-
New Sickle Cell Disease Research Shows Improved Patient OutcomesEmerging therapies and promising new ins...
-
Sickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
-
BCL11A-based gene therapy for sickle cell disease passes key preclinical testA precision-engineered gene therapy viru...
-
Gene Therapy for Blood DisordersIn the context of intense scrutiny over ...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.