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INFORMATION FOR

Farzana Pashankar

MD, MRCP, MBBS
Professor of Pediatrics (Hematology/Oncology); Fellowship Director, Pediatric Hematology & Oncology Program; Director, Solid Tumor Program, Pediatric Hematology & Oncology Program; Children's Oncology Group Site PI, Pediatric Hematology & Oncology Program

Research Summary

My areas of interest include Sickle cell disease and Solid Tumors especially germ cell tumors and rare cancers. In sickle cell disease, my research has focused on a variety of areas including identifying the prevalence and risk factors of various complications, management of these complications and quality improvement projects to decrease the burden of disease and improve quality of life.

In oncology, my research activities have focused on collaborative studies through the Children's Oncology Group ( COG) in germ cell tumors and rare tumors. I serve on the steering committees for Germ Gell Tumors and Rare Tumors at COG. I am a member of the Malignant Germ Cell Tumor International Collaborative. Through this collaborative, we have merged 25 years of clinical trial data from US and UK pediatric and adult germ cell clinical trials. This has helped us redefine risk stratification and conduct several analyses. I took leadership of analyzing the data on ovarian immature teratomas. Ovarian immature teratomas are a rare malignancy. There is significant difference in treatment approach between the adult and pediatric groups. In pediatrics, patients are primarily treated with surgery alone, whereas adults receive chemotherapy for all ovarian IT, except Stage I, grade 1 patientsI analyzed the pediatric and adult clinical trial data, and found that grade was the most important risk factor for relapse. More importantly, I found that despite a wide difference in treatment approaches, between adults and pediatrics, the outcomes were similar, bringing up the question of the necessity of adjuvant chemotherapy for ovarian IT.

Specialized Terms: Clinical trials in Pediatric Oncology; Sickle cell anemia

Extensive Research Description

1. Sickle Cell Disease

My research has focused on a variety of areas including identifying the prevalence and risk factors of various complications, management of these complications and quality improvement projects to decrease the burden of disease and improve quality of life.

In the 1990’s, a landmark study from the NIH reported that pulmonary hypertension occurred in a third of adults and was associated with increased mortality. There was limited data in children. I thus designed the first prospective screening study and identified that elevated pulmonary artery pressures (PAP) occur in 30% of children, and is associated with hypoxia, hemolysis and a high platelet count. This was published in Pediatrics and has been widely quoted. Although elevated PAP occurred in children, there was no data on the long-term outcome and possible reversibility of this complication. I conducted a single institution pilot study to determine if treatment with Hydroxyurea, an antihemolytic agent could reverse elevated PAP in children. This study showed that elevated PAP was reversible with hydroxyurea and was published in British Journal of Hematology. Due to the exciting results from our single center pilot, we secured CTSA funding and conducted a multicenter study. This study showed that hydroxyurea improved oxygenation in SCD and had a trend in decreasing PAP.

Working with a medical student, we studied the association of PAP with other complications and found that elevated PAP was associated with early signs of nephropathy. This was the first study describing this association.

Over the years I have collaborated with colleagues at Yale and through the New York Connecticut Consortium and New England Pediatric Sickle Cell consortium to study other complications such as retinopathy, AVN, obesity and determine genetic determinants of hydroxyurea response and look at barriers to hydroxyurea use.

Recently I have focused on quality improvement projects to improve pain management at home and decrease ED visits and hospitalizations. This has been very successful in reducing hospitalizations and improving the quality of life for these families. This work was published and has been adopted by many institutions.

Current Studies and Future Direction:

1. Understanding and Addressing the Social Determinants of Health for Families of Children with Sickle Cell Anemia within Pediatric Hematology

2. Rare Tumors

I was the chair of the Infrequent Tumor Subcommittee at COG till 2020. We developed and published guidelines for several rare tumors and have recently completed two international clinical trials, on adrenocortical carcinoma and nasoparyngeal carcinoma.

3. Germ Cell Tumors

In germ cell tumors, my research has focused on redefining management of immature teratomas (IT) and clinical trial development. The management of ovarian IT is controversial. In children, ovarian IT is treated with surgery alone, whereas adults treat all patients with chemotherapy except those with Stage I, grade 1 tumors. I analyzed pediatric and adult clinical trial data from the MaGIC database and found that grade was the most important risk factor for relapse. In patients with grade 3 tumors, the outcome was similar between the pediatric and adult cohort despite a marked difference in treatment, questioning the role of chemotherapy. Since that initial analyses, I have published other reports showing chemo unresponsiveness of ovarian IT. We have also diligently worked with the adult gynecologic oncology community to move away from using chemotherapy postoperatively in ovarian IT.

Our efforts have been somewhat successful and while designing the current open trial AGCT1531 in conjunction with Gynecology Oncology Group, they agreed to observation alone not only for stage I, grade 1 tumors but also for Stage I, grade 2 and 3 tumors. This is a major change and has the potential to spare chemotherapy to many adult women if the trial proves our hypothesis. We are currently working on an amendment to expand surveillance alone to all patients with ovarian IT, irrespective of stage and I am also organizing a pathology consensus conference to standardize grading of IT.

Another area that I have been actively involved in is clinical trial development at Children’s Oncology Group (COG). Currently we have two clinical trials open – I am the Vice Chair on an international trial for low and intermediate risk germ cell tumors and am the Chair for all US sites for an international trial for poor risk germ cell tumors run by Australia New Zealand Urogenital group, that is open in the US through National Clinical Trials Network.

At Yale, I direct the solid tumor program, and have been actively working to bring innovative industry sponsored trials in addition to trial through COG to broaden our trial portfolio.



Coauthors

Research Interests

Anemia, Sickle Cell; Echocardiography; Hematology; Hypertension, Pulmonary; Medical Oncology; Pediatrics

Selected Publications

Clinical Trials

ConditionsStudy Title
Brain and Nervous System; PediatricsA Phase 3 Randomized Study of Selumetinib Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Brain and Nervous SystemA Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated With BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
PediatricsA Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations
PediatricsPrOspective Non-interventional Study in Patients With Locally Advanced or Metastatic TRK Fusion Cancer Treated With Larotrectinib
Supportive Care; Pediatrics; PreventionStepByStep: A Randomized Trial of a Mobile Health and Social Media Physical Activity Intervention Among Adolescent and Young Adult Childhood Cancer Survivors
Brain and Nervous System; PediatricsA Target Validation/Phase1 Study of BGB-290 in Combination With Temozolomide in Adolescent and Young Adult IDH1/2 Newly Diagnosed and Recurrent Mutant Gliomas
Brain and Nervous System; PediatricsA Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)
Leukemia, not otherwise specified; Leukemia, other; PediatricsA Phase 3 Randomized Trial of Inotuzumab Ozogamicin (NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy
PediatricsA Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab With or Without Eflornithine (DFMO) in Children With Relapsed, Refractory or Progressive Neuroblastoma
LiverPediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Leukemia, not otherwise specified; Leukemia, otherA Phase IB/II Study of Venetoclax (ABT-199) in Combination With Liposomal Vincristine in Patients With Relapsed or Refractory T-Cell or B-Cell Acute Lymphoblastic Leukemia
Brain and Nervous SystemA Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)
Bones and Joints; Kidney; Soft Tissue; PediatricsPhase 3 Accelerated BEP: A Randomised Phase 3 Trial of Accelerated Versus Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumors
Other Hematopoietic; Pediatrics; Sickle Cell DisordersA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime
Soft TissueA Phase 2 Study of Anti-PD-L1 Antibody (Atezolizumab) in Alveolar Soft Part Sarcoma
Lymphoid LeukemiaA Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia
Other Male Genital; OvaryActive Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
Bones and Joints; Brain and Nervous System; Eye and Orbit; Hodgkin's Lymphoma; Kidney; Leukemia, other; Liver; Lymphoid Leukemia; Myeloid and Monocytic Leukemia; Non-Hodgkin's Lymphoma; Other Digestive Organ; Other Endocrine System; Other Female Genital; Other Male Genital; Other Respiratory and Intrathoracic Organs; Other Skin; Other Urinary; Ovary; Small Intestine; Soft Tissue; PediatricsThe Project: Every Child Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
Other Respiratory and Intrathoracic Organs; PediatricsEffects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment (HEART)
Brain and Nervous System; PediatricsResponse and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma