Hematology/Oncology Research

Cancer is the leading non-accidental cause of childhood death. Yet, thanks to research, since the 1970s childhood cancer deaths have declined 35 percent, and in the case of leukemia by 50 percent. However, the battle is not over. Each year 25 percent of children diagnosed with cancer will die of their disease.

Statistics like these fuel a passion to find cures within physicians and scientists of the Division of Hematology/Oncology of Children's Hospital of Pittsburgh of UPMC. The division has extensive research interests in clinical treatment for and basic mechanisms underlying cancer, ultimately aimed at improving clinical therapies. Its commitment to advancing the science of hematology and oncology includes basic research extending into the areas of stem cell biology and molecular oncology.

With so much at stake, Children’s Hematology/Oncology team ranks first in sheer volume of clinical studies within the divisions of Children’s Hospital of Pittsburgh. This is due, in part, to its strong relationship with the Children’s Oncology Group, the only pediatric oncology clinical trials group funded by the National Cancer Institute and National Institutes of Health (NIH). At Children’s, clinical trials are underway related to bone marrow transplantation; treatment of sickle cell disease; cancer control and late-effects in oncology. An ongoing oncology study at Children’s examines incidence of graft-versus-host disease following unrelated donor stem cell transplantation. Children’s is the only institution in the region participating in Phase 1 clinical trials through the Children’s Oncology Group and the Pediatric Brain Tumor Consortium. These important and innovative studies of new cancer treatments are critical to our progress in achieving the goal of curing all children with cancer.

Ongoing NIH-funded oncology projects include the study of critical biochemical events that control the growth, maturation and death of cells that cause leukemia and the study of a family of genes that cause cancers such as Burkitt’s lymphoma and neuroblastoma.

As leaders in their field, Children’s Hematology/Oncology scientists have access to the most advanced tools and resources. Genetic microarray technology, for example, is being utilized in a study on the mechanisms that cause cells to become cancerous or resistant to anti-cancer drugs. This cutting-edge technique, based on results of the Human Genome Project, allows one to measure simultaneously how much protein is being made by more than 5,000 different genes in a single cell at any given time.

The Hematology/Oncology team has not limited itself to the scientific studies of childhood cancers, but also addresses the special medical and psychosocial needs of cancer patients. Through this broad-spectrum approach Children’s Division of Hematology/Oncology continues to forge ahead in its efforts to explore, diagnose, treat and cure childhood cancers.

Physicians at Children’s Hospital are also active in clinical care and research in diseases of blood other than cancer. Sickle cell disease is a serious chronic illness of the red blood cell that is complicated by anemia, stroke, lung complications and decreased quantity and quality of life. The sickle cell program at Children’s Hospital is one of eight sites around the world that are participating in a clinical trial to determine the prevalence of pulmonary hypertension a serious complication involving the heart and lung. Patients found to have pulmonary hypertension will be treated in a randomized double-blind study of a drug called sildenafil. This groundbreaking study is expected to provide information on a serious and little understood complication of this disease.

Researchers at Children’s Hospital are also leading a study to cure sickle cell disease by a gentler type of bone marrow transplant. This approach has the potential to make this curative treatment to many patients to whom this was not previously available. Research in sickle cell disease also includes approaches on how best to provide comprehensive care, education and counseling to a population that disproportionately affects individuals who belong to minority, underserved groups, by using a comprehensive "medical home" approach and the use of medical information technology.


Doctors

Clinical Studies

Acute Lymphoblastic Leukemia and B-Lineage Lymphoma Treatment via Risk-Adapted Chemotherapy – Phase III

Risk-Adapted Chemotherapy in Treating Younger Patients with Newly Diagnosed Standard-Risk B-Acute Lymphoblastic Leukemia or localized B-Lineage Lymphoblastic Lymphoma

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Acute Lymphoblastic Leukemia Cell Banking Study

Proper Protocols for Collecting and Banking Relapsed Acute Lymphoblastic Leukemia Research Specimens

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Acute Myeloid Leukemia trial using Bortezomib and Sorafenib – Phase III

Trial for Patients with de novo Acute Myeloid Leukemia using Bortezomib and Sorafenib for Patients with High Allelic Ratio FLT3/ITD

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Acute Promyelocytic Leukemia Treatment using Arsenic Trioxide and All Trans-Retinoic Acid – Phase III

Study for Patients with Newly Diagnosed Acute Promyelocytic Leukemia using Arsenic Trioxide and All Trans-Retinoic Acid

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Anaplastic Large Cell Lymphoma Treatment with Brentuximab Vedotin or Crizotinib – Phase II

Brentuximab Vedotin or Crizotinib in Combination with Chemotherapy for Newly Diagnosed Patients with Anaplastic Large Cell Lymphoma

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Brain Tumor Memory Study – Phase II

Trial of Modafinil to Improve Neurocognitive Deficits in Children Treated for a Primary Brain Tumor

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Central Nervous System Germ Cell Tumor Treatment with Chemotherapy and Radiation – Phase II

Response-Based Radiation Therapy for Patients with Localized Central Nervous System Germ Cell Tumors

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Central Nervous System Tumor Treatment with PD-0332991 – Phase I

A Study of CDK 4-6 Inhibitor PD-0332991 in Children with Recurrent, Progressive or Refractory Central Nervous System Tumors

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Craniopharyngioma Treatment with PEGIntron – Phase II

Study of Peginterferon alfa-2b (PEGIntron) for Pediatric Patients with Unresectable or Recurrent Craniopharyngioma

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DIPG Treatment with MK-1775 and Local Radiation – Phase I

MK-1775 and Local Radiation Therapy in Treating Younger Patients With Newly Diagnosed Diffuse Intrinsic Pontine Gliomas

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Ependymoma Treatment with Maintenance Chemotherapy – Phase III

Trial of Post-Radiation Chemotherapy in Patients with Newly Diagnosed Ependymoma

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Ependymoma Vaccine Trial – Pilot

Treatment for Recurrent Ependymomas in Children Using HLA-A2 Restricted Tumor Antigen Peptides in Combination With Imiquimod

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Ewing Sarcoma Tissue Banking Study

A Children’s Oncology Group Protocol for Collecting and Banking Ewing Sarcoma Specimens

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Ewing Sarcoma Treatment with Combination Chemotherapy Including Ganitumab – Phase II

Adding the IGF-1R Monoclonal Antibody Ganitumab to Multiagent Chemotherapy for Patients with Newly Diagnosed Metastatic Ewing Sarcoma

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Ewing Sarcoma Treatment with Combination Chemotherapy Including Topotecan – Phase III

Adding Vincristine-topotecan-cyclophosphamide to Standard Chemotherapy in Initial Treatment of Non-metastatic Ewing Sarcoma

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Gliomas (Low-Grade) Vaccine Study – Phase II

Vaccinations with HLA-A2-Restricted Glioma Antigen-Peptides in Combination with Poly-ICLC for Children with Recurrent Unresectable Low-Grade Gliomas (LGG)

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Glioma Treatment with Selumetinib (AZD6244) – Phase II

Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma

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Glioma Treatment with Veliparib, Radiation and Temozolomide – Phase I/II

ABT-888, an Oral Poly (ADP-ribose) Polymerase Inhibitor, and Concurrent Radiation Therapy, Followed by ABT-888 and Temozolomide, in Children with Newly Diagnosed Diffuse Pontine Gliomas

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Gliomas Vaccine Study – Pilot

Treatment of Newly Diagnosed Brainstem Gliomas and Non-Brainstem High-Grade Gliomas, Recurrent Low-Grade and High-Grade Gliomas

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High-Risk Acute Lymphoblastic Leukemia Treatment with Combination Chemotherapy – Phase III

Trial for Newly Diagnosed High Risk B-precursor Acute Lymphoblastic Leukemia, Testing Clofarabine in the Very High Risk Stratum

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High-Risk Medulloblastoma Treatment with Chemotherapy and Radiation – Phase III

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

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Hodgkin Lymphoma Treatment with Brentuximab Vedotin – Phase III

Study of Brentuximab Vedotin for Newly Diagnosed High-Risk Classical Hodgkin Lymphoma in Children and Adolescents

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Hodgkin Lymphoma Treatment with Brentuximab Vendotin and Gemcitabine – Phase I/II

Brentuximab Vedotin in Combination with Gemcitabine for Pediatric and Young Adult Patients with Relapsed or Refractory Hodgkin Lymphoma

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Medulloblastoma and PNET Therapies with Temozolomide, Irinotecan, and Bevacizumab – Phase II

Temozolomide with Irinotecan versus Temozolomide, Irinotecan plus Bevacizumab for Recurrent/Refractory Medulloblastoma/CNS PNET of Childhood, A COG Randomized Phase II Screening Trial

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Medulloblastoma Treatment with Carboplatin and Isotretinoin – Phase III

Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET Patients

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M0 Medulloblastoma Treatment in Very Young Children – Phase I

Study of Eribulin Mesylate, a Novel Microtubule Targeting Chemotherapeutic Agent, in Children with Refractory or Recurrent Solid Tumors including Lymphomas

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M0 Medulloblastoma Treatment in Young Children – Phase II

Study for the Treatment of Non-Metastatic Nodular Desmoplastic Medulloblastoma in Children Less Than 4 Years of Age

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Non-Hodgkin Lymphoma or B-Cell Leukemia Evaluation of Rituximab– Phase II/III

Intergroup Trial for Children or Adolescents with B-cell Non-Hodgkin Lymphoma or Mature B-cell Leukemia: Evaluation of Rituximab Efficacy and Safety in High Risk Patients

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Osteosarcoma Response to Dinutiximab and Sargramostim – Phase II

Human-Mouse Chimeric Anti-Disialoganglioside Monoclonal Antibody ch14.18 (Dinutuximab) in Combination with GM-CSF (Sargramostim) in Patients with Recurrent Osteosarcoma

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Osteosarcoma Tissue Banking Study

A Children’s Oncology Group Protocol for Collecting and Banking Osteosarcoma Specimens

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Pediatric Tumors and Specimen Study

A Children’s Oncology Group Protocol for Collecting and Banking Pediatric Specimens Including Rare Pediatric Tumors

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Pilocytic Astrocytoma and Optic Pathway Glioma Treatment with Lenalidomide – Phase II

Trial of Lenalidomide in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas

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Plexiform Neurofibromas Treatment with PEG-Intron - Phase II

Peginterferon alfa-2b in Treating Young Patients with Unresectable, Sympotomatic or Life-Threatening Plexiform Neurofibromas Associated with Neurofibromatosis Type 1

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Sickle Cell Acute Chest Syndrome Treatment with Prasugrel – Phase III

A Comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease

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Sickle Cell Disease Treatment with BMT and Conditioning (STRIDE) – Phase II

A Study of Hematopoietic Stem Cell Therapy for Young Adults with Severe Sickle Cell Disease

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Sickle Cell Medication Management Study

Patient-Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy with Hydroxyurea in Patients with Sickle Cell Disease

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Sickle Cell Pain Crisis Management Study (SUSTAIN) – Phase II

Study to Assess Safety and Efficacy of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients with Sickle Cell-Related Pain Crises (SUSTAIN)

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Sickle Cell Vaso-Occlusive Pain Crisis Treatment Study (EPIC) – Phase II

Evaluation of Purified Poloxamer 188 (MST 188) in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)

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Soft Tissue Sarcoma Treatment with Preoperative Radiation and Pazopanib (PAZNTIS Trial) – Phase II/III

Pazopanib Neoadjuvunt Trial on Non-Rhabdomyosarcoma Soft Tissue Sarcomas (PAZNTIS): A Trial of Preoperative Chemoradiation or Preoperative Radiation Plus or Minus Pazopanib

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Solid Tumor and Lymphoma Treatment with PF-02341066 – Phases I and II

PF-02341066, an Oral Small Molecule Inhibitor of Anaplastic Lymphoma Kinase (ALK) and c-MET, in Children with Relapsed/Refractory Solid Tumors and Anaplastic Large Cell Lymphoma

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Solid Tumor or ALCL Treatment with Crizotinib and Conventional Chemotherapy – Phase I

Crizotinib in Combination with Conventional Chemotherapy for Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma

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Solid Tumor Treatment with MK-1775 – Phase I/II

MK-1775 in Combination with Oral Irinotecan in Children, Adolescents, and Young Adults with Relapsed or Refractory Solid Tumors

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Solid Tumor Treatment with Ramucirumab – Phase I

A Study of Ramucirumab, a Human Monoclonal Antibody Against the Vascular Endothelial Growth Factor-2 Receptor in Children with Refractory Solid Tumors, Including CNS Tumors

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T-cell Lymphoblastic Leukemia and Lymphoma Treatment with Bortezomib – Phase III

Bortezomib on a Modified Augmented BFM (ABFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LLy)

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Transient Myeloproliferative Disorder Blood Study

Biology Study of Transient Myeloproliferative Disorder in Children with Down Syndrome

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Tumor and Acute Lymphoblastic Leukemia Treatment with BMN 673 and Temozolomide – Phase I/II

BMN 673, an Oral Poly Polymerase Inhibitor, Plus Temozolomide in Children with Refractory or Recurrent Malignancies

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Tumor Treatment with Nivolumab with and without Ipilimumab – Phase I/II

Nivolumab in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors as a Single Agent and in Combination with Ipilimumab

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Wilms Tumor Treatment with Combination Chemotherapy and Surgery – Phase III

Treatment for Patients with Bilateral, Multicentric, or Bilaterally-Predisposed Unilateral Wilms Tumor

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