Anne Stone   Assistant Professor of Pediatrics

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1. Do Preexisting Lung Function Abnormalities Predict Acute Pulmonary Impairment in Children Undergoing Hematopoietic Cell Transplant for Acute Leukemia or Myelodysplastic Syndrome? This project aims to determine the relationship between pre-existing obstructive or restrictive defects and short-term pulmonary outcomes after HCT by measuring spirometry, airway responsiveness, lung volumes, and diffusion capacity prospectively on children with acute leukemia and MDS before and after HCT. Children undergoing transplant at MSKCC are referred for study enrollment and lung function testing prior to transplant with follow-up lung function testing 6 months and 1 year.

2. Long-term Pulmonary Outcomes in Survivors of Advanced Stage Neuroblastoma. The overall survival rate for neuroblastoma patients with advanced stage disease is approximately 30% at 5 years. Myeloablative therapy with autologous hematopoietic cell transplant and other newer treatments have led to improved survival for a subset of these children. These survivors confront multiple late toxicities of intensive therapy received at a young age. This study assesses PFTs of pediatric survivors of advanced stage NB who are followed in the Long-term Follow-up clinic at Memorial Sloan Kettering Cancer Center. The study will examine late pulmonary outcomes among advanced stage NB survivors and help guide future research in the field of pediatric cancer survivorship.

3. Pulmonary Function Testing Abnormalities in Thalassemia Major and the Role of Impulse Oscillometry. Multiple prior investigations have demonstrated functional pulmonary abnormalities among children with thalasssemia major (TM). The majority of reports have demonstrated restrictive lung disease that increases in prevalence with age and transfusional iron burden. This study aims to measure lung function by spirometry, plethysmography, and impulse oscillometry among a large cohort of children with TM followed at the WCMC TM clinic. Chart review will identify potential risk factors for pulmonary abnormalities.

4. Severe asthma with fungal sensitization (SAFS) in children: identification of genetic risk factors and characterization of biomarkers. Patients with difficult to manage asthma represent a small percentage of the total asthma population (estimated to be 5-10%) but account for the majority of poor outcomes. Treatment for such patients is limited and current recommendations focus on high dose inhaled or systemic corticosteroids which can lead to significant side effects. There is increasing interest in the role of fungal infections in severe asthma. Fungi have been linked to severe asthma in numerous studies and anti-fungal treatment has been reported to improve clinical symptoms for some asthmatics, including those with severe asthma with fungal sensitization (SAFS). This multi-center pilot observational study aims to determine the prevalence of SAFS in our patient population and to determine whether genetic mutations are associated with SAFS and allergic inflammation. Patients are approached for possible study participation if they have poorly controlled asthma and are undergoing standard of care blood tests. An additional sample of serum is taken for genetic tests and tests for inflammatory markers if subjects enroll.


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