Farzana Pashankar, MD, MRCP

Associate Professor of Pediatrics (Hematology / Oncology)

Research Departments & Organizations

Pediatrics: Pediatric Hematology & Oncology

Yale Cancer Center: Developmental Therapeutics

Office of Cooperative Research

Research Interests

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

Research Summary

My areas of interest include Sickle cell disease and Rare Tumors especially germ cell tumors and infrequent cancers. In sickle cell disease, my focus has been on studying the prevalence, risk factors and potential treatment options for pulmonary hypertension in children with sickle cell disease.

In oncology, my research activities have focused on collaborative studies through the Children's Oncology Group in germ cell tumors and infrequent cancers. I am the Chair of the Infrequent tumor Subcommittee at COG. We developed and published guidelines for several rare tumors and have recently completed two international clinical trials, on adrenocortical carcinoma and nasoparyngeal carcinoma. 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

Pulmonary hypertension occurs in one third of adults with SCD disease and is an independent risk factor for mortality. There was limited data in children. I thus designed a prospective screening study. This study identified that elevated pulmonary artery pressures (PAP) occurs in 30% of children, and is associated with hypoxia, hemolysis and a high platelete count. This was the first prospective screening study in children, and showed that similar to adults, children develop elevated PAP. This was published in Pediatrics, and has been widely quoted.

Our screening study showed that children develop elevated PAP. However there was no data on the long term outcome and possible reversibility of this complication. I designed 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 conducting a multicenter study. This study was limited by sample size, and showed a trend towards decrease in PAP with hydroxyurea. The study did show that hydroxyurea improved oxygenation in SCD.

Working with a medical student, we looked at the association of nephropathy with elevated PAP, and found that elevated PAP was associated with early signs of nephropathy, in form of proteinuria. This was the first study describing this association, and was published.

Current Studies and Future Direction

1. We have an ongoing longitudinal follow up study of elevated PAP in children with SCD. This study will help us determine if children with elevated PAP, develop the irreversible PHT in adults.

2. Emotional Intelligence and Health Related Quality of Life Outcomes in Children with Sickle Cell Anemia.

3.Prevalence and Progression of Retinopathy in children with Sickle Cell Disease

4. Sickle Cell Nepal- A Pilot research and education project for sickle cell disease in Western

2. Rare Tumors

I am the chair of the Infrequent Tumor Subcommittee at COG. 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

1 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 patients

I 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.

This paper has been accepted in Cancer

Future direction: In the soon to be opened low/intermediate risk germ cell trial, all pediatric and adult patients with ovarian IT will be observed.


Clinical Trials

Conditions Study Title
Liver Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
Brain and Nervous System A Phase 2 Study of Veliparib (ABT-888) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients With Newly Diagnosed High-Grade Glioma (HGG) Without H3 K27M or BRAFV600E Mutations
Brain and Nervous System A 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, Pediatrics Phase 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
Pediatrics Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults With Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors
Leukemia, other A Groupwide Pilot Study to Test the Tolerability and Biologic Activity of the Addition of Azacitidine (NSC# 102816) to Chemotherapy in Infants With Acute Lymphoblastic Leukemia (ALL) and KMT2A (MLL) Gene Rearrangement
Other Hematopoietic, Pediatrics A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime
Soft Tissue A Non-Randomized, Open-Label, Phase 2 Study of Trametinib in Patients With Unresectable or Metastatic Epithelioid Hemangioendothelioma
Pediatrics A Phase 1/2 Study of VX15/2503 in Children, Adolescents, or Young Adults With Recurrent or Relapsed Solid Tumors
Bones and Joints, Brain and Nervous System, Leukemia, not otherwise specified, Leukemia, other, Lymphoid Leukemia NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Soft Tissue A Phase 2 Study of Anti-PD-L1 Antibody (Atezolizumab) in Alveolar Soft Part Sarcoma
Lymphoid Leukemia A 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
Hodgkin's Lymphoma, Pediatrics Risk-based, Response-adapted, Phase II Open-label Trial of Nivolumab + Brentuximab Vedotin (N + Bv) for Children, Adolescents, and Young Adults With Relapsed/Refractory (R/R) CD30 + Classic Hodgkin Lymphoma (cHL) After Failure of First-line Therapy, Followed by Brentuximab + Bendamustine (Bv + B) for Participants With a Suboptimal Response (CheckMate 744: CHECKpoint Pathway and Nivolumab Clinical Trial Evaluation)
Anus, Breast - Female, Breast - Male, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Pediatrics, Cervix, Urinary Bladder A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
Other Male Genital, Ovary Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
Anus, Bones and Joints, Brain and Nervous System, Breast - Female, Breast - Male, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Hodgkin's Lymphoma, Kaposi's sarcoma, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung, Melanoma, skin, Mycosis Fungoides, 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, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Pediatrics, Cervix, Urinary Bladder A Phase I/II Study of Pembrolizumab (MK-3475) in Children With Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma (KEYNOTE-051)
Anus, Bones and Joints, Brain and Nervous System, Breast - Female, Breast - Male, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Hodgkin's Lymphoma, Kaposi's sarcoma, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung, Lymphoid Leukemia, Melanoma, skin, 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, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Cervix, Urinary Bladder NCI-MATCH: Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors or Lymphomas
Brain and Nervous System, Pediatrics A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma
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, Pediatrics The Project: Every Child Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
Other Respiratory and Intrathoracic Organs, Pediatrics Effects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment (HEART)

Selected Publications

See list of PubMed publications

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