Originally published on September 10, 2013.

Dr. Mayer and Ms. Battista were awarded funding by Cure SMA for one of four new clinical care focused projects. The project is being conducted at the Children’s Hospital of Philadelphia. This award for $50,000 will fund a pilot study to assess the decision making process by parents of children with SMA on clinical care options.  

Project Description:

Decisions Related to Goals and Limitations of Care and the Challenges in Making Them for Parents of Children with SMA

Objective: The aim of the proposed study is to assess the possibility of conducting research with parents of children with SMA, as has been done in populations of parents of children with other serious illness, and to assess parents’ decision-making processes, perceptions about parenting, and levels of depression and anxiety as changes in their children’s medical status occur.
Research Strategy: We propose to conduct a study involving 50 parents of children
Significance of the Project: To our knowledge, this proposed study will provide the richest data set available involving perspectives of parents’ of children with SMA-1 and SMA-2, and will thereby provide the basis for a larger study and ultimately the design of a supportive care intervention trial.

Meet Dr. Mayer:

Who are you?
Dr. Oscar Henry “Hank” Mayer is an Associate Professor of Clinical Pediatrics at the Perelman School of Medicine at the University of Pennsylvania School of Medicine and is an Attending Physician in the Division of Pulmonology and on the Pediatric Advanced Care Team at the Children’s Hospital of Philadelphia (CHOP).  He has been active in the clinical management of Children with SMA and the Pulmonologist within the Neuromuscular Program at CHOP since 2001.  He has had a long standing interest in optimizing the clinical care of patients with SMA both in direct therapy focused on medical treatment and parental/family support in decision making related to limitations of care and quality of life.

How did you first become involved in SMA research?
Dr. Mayer has explored a variety of approaches to assess respiratory status in patients with SMA and has been doing work to assess the components of progressive thoracospinal disease.  He has spent the last two years working with the current research group on approaches to assess decision making on limitations of care and end of life, and is excited about the possibility to explore this very important
topic.

What is your current role in SMA research?
Dr. Mayer will be the Principal Investigator in this study and will be involved in data analysis, manuscript preparation and general study oversight.

Meet Ms. Battista:

Who are you?
Vanessa Battista, RN, MS, CPNP, CCRC, currently works as a Pediatric Nurse Practitioner (PNP) with the Pediatric Advanced Care Team (PACT) at The Children’s Hospital of Philadelphia. She holds a B.A. in psychology from Boston College, a B.S. and M.S. from Columbia University School of Nursing, and a certificate in Pastoral Ministry from the Boston College School of Theology and Ministry. Initially, Vanessa worked as a clinical research coordinator, RN, and then PNP at the SMA Research Center at Columbia University Medical Center in NY, and then practiced as a PNP at the SMA Center at Children’s Hospital Boston, before relocating to Philadelphia. Vanessa’s interests lie in providing interdisciplinary care to children/adolescents and families living with chronic conditions. She lectures and teaches locally and nationally and serves on various advisory boards. Her research interests lie in clinical aspects of caring for children and families living with SMA. She is also a member of the Cure SMA Medical Advisory Council (MAC).

How did you first become involved in SMA research?
Vanessa has been caring for children and families living with SMA for the past ten years and has participated in various aspects of clinical research with this population. She first became involved in research as a certified clinical research coordinator (CCRC) and is looking forward to being a part of the initial studies to explore clinical aspects of living with SMA, this time in the role of Co-Investigator.

What is your current role in SMA research?
Vanessa will serve as a Co-Investigator on this study aiming to explore parents’ decision making processes as they do their best to care for and parent their children with SMA.  She will be involved in subject identification, study oversight, and manuscript preparation.

Cure SMA has been funding critical research to develop a treatment and cure for the disease since 1984, along with providing important resources and support for families affected by SMA. In 2013, we launched a new program to fund care research to drive improvements in patient care in spinal muscular atrophy. This program is focused on improving care and the quality of life for SMA patients.

The results of funded projects will build an evidence base and demonstrate measurable, positive effects on the clinical management and lives of patients with SMA. These results will then be used to provide:

•    Educational programs for professional medical providers, such as the Cure SMA CME day
•    New family-focused care publications, such as the Cure SMA Care Series Booklets
•    Peer reviewed journal publications to influence insurance coverage