Division Data Summary

Research and Training Details

Number of Faculty12
Number of Joint Appointment Faculty4
Number of Research Fellows1
Number of Research Students1
Number of Support Personnel54
Direct Annual Grant Support$917,385
Direct Annual Industry Support$8,624
Peer Reviewed Publications39

Clinical Activities and Training

Number of Clinical Staff4
Number of Clinical Fellows4
Inpatient Encounters9,133
Outpatient Encounters3,816

Division Photo

Bone Marrow Transplantation and Immune Deficiency
Left to Right: R Marsh, K Myers, S Jodele, A Kumar, M Jordan, A Filipovich, S Davies, M Grimley, P Mehta, S Joshi

Significant Publications

Laskin BL, Goebel J, Davies SM, Khoury JC, Bleesing JJ, Mehta PA, Filipovich AH, Paff ZN, Lawrence JM, Yin HJ, Pinkard SL, Jodele S. Early clinical indicators of transplant-associated thrombotic microangiopathy in pediatric neuroblastoma patients undergoing auto-SCT. Bone Marrow Transplant. 46:682-9, 2011.
Marsh RA, Madden L, Kitchen BJ, Mody R, McClimon B, Jordan MB, Bleesing JJ, Zhang K, Filipovich AHXIAP deficiency: a unique primary immunodeficiency best classified as X-linked familial hemophagocytic lymphohistiocytosis and not as X-linked lymphoproliferative disease. Blood. 116:1079-82. 2010.
Marsh RA, Vaughn G, Kim MO, Li  D, Jodele  S, Joshi  S, Mehta  PA, Davies  SM, Jordan MB, Bleesing JJ, Filipovich  AH. Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. Blood. 116:5824-31. 2010.
Smith AR, Majhail NS, MacMillan ML, DeFor TE, Jodele S, Lehmann LE, Krance R, Davies SM. Hematopoietic cell transplantation comorbidity index predicts transplantation outcomes in pediatric patients. Blood. 117:2728-34. 2011.

Division Collaboration

Immunobiology » L. Grimes
MEIS1 and MLL-fusion leukemia: Research project investigating the role of MEIS1 in MLL-fusion leukemia using transgenic mice. (A. Kumar)
Experimental Hematology and Cancer Biology » J. Mulloy
Animal models of human leukemia: Research project aimed at developing xenograft mouse models of human leukemia using primary patient derived material. (A. Kumar)
Experimental Hematology and Cancer Biology » J. Mulloy; J. Cancelas
The molecular origin of monosomy 7 in very young children.  Translational research project exploring the causes of monosomy 7.  (P. Mehta, SM Davies)
Experimental Hematology and Cancer Biology » N. Ratner
MEIS1 and MPNSTs: Research project aimed at understanding the role of MEIS1 in malignant peripheral nerve sheath tumors (MPNST). (A. Kumar)
Nephrology; Biostatistics & Epidemiology » J. Goebel; B. Dixon; P. Devarajan; J. Khoury
A Prospective Analysis of Clinical and Biochemical Markers for Pediatric Stem Cell Transplant-Associated Thrombotic Microangiopathy. Study open and more than half the needed patients enrolled.  Preliminary data generated and grant applications submitted. (J. Bleesing, SM. Davies, A. Filipovich, R. Harris, S. Jodele, M. Jordan, S. Joshi, A. Kumar, R. Marsh, P. Mehta)
Critical Care; Radiology; Nephrology » H. Wong; S. Poynter; W. Ball; J. Leach; J. Goebel; M. Mitsnefes
PRES working group: To identify patients at risk for PRES, outline uniform evaluation and therapy guidelines. (S. Jodele, SM Davies)
Nephrology » J. Goebel; BL Laskin
Jodele S Davies SM, Lawrence J, Mehta PA, Bleesing JJ, Filipovich AH, Marsh RA. Blood, and Not Urine, BK Viral Load Predicts Renal Outcome in Children with Hemorrhagic Cystitis following Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2011
Nephrology » J. Goebel; BL Laskin
Small vessels, big trouble in the kidneys and beyond: hematopoietic stem cell transplantation-associated thrombotic microangiopathy. (S Jodele, SM Davies).
Critical Care » R. Chima; D. Wheeler
BMT-PICU risk score index working group: To determine outcome predictors for BMT patients that are treated in PICU. To validate BMT-PICU risk score at CCHMC and later at collaborating institutions. (S Jodele, SM Davies)
Critical Care » R. Chima; D. Wheeler
Paper describing excellent outcomes for children admitted to PICU post-transplant admitted to PICU.  (S Jodele, SM Davies)
Surgery; Gastroenterology » J. Nathan; G. Tiao; M. Alonzo; N. Yazigi ; S. Kocoschis
Incidence of acute and chronic graft versus host disease and donor T-cell chimerism after small bowel or combined organ transplantation.  In press in the Journal of Pediatric Surgery.
Surgery ; Dermatology » R. Azizkhan ; A. Lucky
Preparation of a stem cell transplant protocol for children with epidermolysis bullosa. (S Jodele and SM Davies).
Genetics; Pathology » T. Smoralek; S. Zimmerman ; R. McMasters
Myers K, Davies SM, Harris RE, Mehta PA. The clinical phenotype of children with Fanconi anemia caused by biallelic FANCD1/BRCA2 mutations. Pediatr Blood Cancer. 2011.

Faculty Members

Stella Davies, MBBS, PhD, MRCP, Professor
Jacob G. Schmidlapp Endowed Chair; Director, Bone Marrow Transplantation and Immune Deficiency
Executive Co-Director, Cancer and Blood Diseases Institute
Director, Division of Bone Marrow Transplantation and Immune Deficiency
Research Interests Blood and Marrow Transplant, Fanconi anemia and other marrow failure disorders, Pharmacogenetics and Pharmacokinetics, Survivorship Research, and BMT for Children with Leukemia.
Jacob Bleesing, MD, PhD, Associate Professor
Research Interests Clinical Investigation of Primary Immunodeficiency Disorders, with emphasis on disorders of immunodysregulation and B-cell disorders
Alexandra Filipovich, MD, Professor
Ralph J. Stolle Chair in Clinical Immunology
Director, Immunodeficiency and
Medical Director, Diagnostic Laboratory
Research Interests Histiocytic disorders,primary immune deficiency disorders and Immunoreconstitution Following Pediatric Stem Cell Transplantation
Michael Grimley, MD, Associate Professor
Research Interests Bone marrow transplantation for children with malignant and non-malignant disorders and treatment of chronic GVHD.
Richard Harris, MD, Professor
Research Interests Transplantation for children with bone marrow failure syndromes and aplastic anemia
Sonata Jodele, MD, Assistant Professor
Research Interests Phase I clinical trials; stem cell transplantation; high risk pediatric malignancies;childhood neuroblastoma, Ewing’s sarcoma, renal complications of transplantation.
Sarita Joshi, MD, Assistant Professor
Research Interests Management of Diamond-Blackfan anemia, transplantation for hemoglobinopathies, and autologous transplantation for malignancy.
Ashish Kumar, MD, PhD, Assistant Professor
Research Interests Etiology of childhood leukemia, transplantation of children with malignancy and primary immune deficiencies.
Rebecca Marsh, MD, Assistant Professor
Research Interests Pathogenesis of HLH with XIAP deficiency, diagnostic laboratory assays, improving outcomes of allogeneic BMT in primary immune deficiencies, use of reduced intensity conditioning regimens.
Parinda Mehta, MD, Assistant Professor
Research Interests Blood and Marrow Transplant, Fanconi anemia and other failure disorders, Pharmacogenetics and Pharmacokinetics
Kasiani Myers, MD, Instructor
Research Interests Blood and Marrow Transplant, Fanconi anemia and other marrow failure disorders, longitudinal studies of hematopoiesis
Janos Sumegi, MD, PhD, Professor
Research Interests Lymphoproliferative disease, Hemophagocytic Lymphohistiocytosis, Usher syndrome, etiology of childhood sarcomas.

Joint Appointment Faculty Members

Mark Johnson, MD, Assistant Professor
Child Psychiatry
Research Interests Psychiatric support of bone marrow transplant patients
Michael Jordan, MD, Assistant Professor
Immunobiology
Research Interests Regulation of the immune response; immunotherapy of cancer, animal models of hemophagocytic disorders.
Mi-Ok Kim, PhD, Associate Professor
Biostatistics & Epidemiology
Ahna Pai, PhD, Assistant Professor
Adherence Psychology in children receiving bone marrow transplantation

Clinical Staff Members

  • Ernest Lawhorn, MD
  • Olive Eckstein, MD
  • Anna Pesok, MD
  • Gregory Wallace, DO

Trainees

  • Hilary Haines, MD, PL-VII, University of Alabama
  • Omar Niss, MD, PL-IV, University of Nebraska Medical Center/Creighton University
  • Chu Ri Shin, MD, PL-VII, Virginia Commonwealth University
  • Jordan Wright, MD, PL-IV, University of Alabama
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Significant Accomplishments

Treatment of HLH

Our Bone Marrow Transplant Program is a leader in diagnosis and treatment of HLH, with children travelling to Cincinnati from across the US and other nations for therapy.  This year Rebecca Marsh, MD, and Lisa Filipovich, MD, published data in the journal Blood about the outcomes of two transplant strategies and identified reduced intensity transplantation as the optimal approach. 

Fanconi Anemia Comprehensive Care Clinic

The Fanconi Anemia Comprehensive Care Clinic continues to expand.  We offer life-long care to more than 150 children and adults with this rare disorder. Most children with Fanconi anemia eventually develop marrow failure and require transplantation.  We have completed an important study of endocrinological abnormalities in this population, led by Susan Rose, MD, Division of Endocrinology.  We also have explored the mechanism of endocrinopathy in a mouse model study lead by Qishen Pang, PhD, Division of Experimental Hematology.  This study showed that oxidant stress causes tissue damage in Fanconi anemia.  Parinda Mehta, MD, has used these data to develop a novel clinical trial using an anti-oxidant food supplement.

Prospective proteomic study of TMA

Transplant associated thrombotic microangiopathy (TMA) is a severe complication of transplantation, commonly leading to organ failure and death.  Sonata Jodele, MD,  in collaboration with the Division of Nephrology, has launched a study of proteomic biomarkers of TMA, identifying possible markers that can predict occurrence of the disorder 10 to 14 days before clinical presentation.  We expect these data to generate new treatment options for this disorder.

Intensive care and bone marrow transplant

Bone marrow transplant is associated with significant morbidity and mortality, and a significant proportion of children will require intensive care.  We have collaborated with Ranjit Chima, MD, and Derek Wheeler, MD, FAAP, to analyze intensive care outcomes in this high-risk population. The data show that 82 percent of children admitted after stem cell transplant survive and leave the ICU.  These outstanding results were presented at the annual meeting of the American Society of Bone Marrow Transplant and the Society for Critical Care Medicine.

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Grants, Contracts, and Industry Agreements

Grant and Contract AwardsAnnual Direct / Project Period Direct

Davies, S

Multicenter Pilot Trial of HSCT Lacking a Genotype Identical Donor
05/01/10-04/30/13$1,920
Antileukemic Effect of NK Cells in HCT for Pediatric AML
R01 CA 12058308/01/07-06/30/12$8,864
Environmental Exposure: Susceptibility Alleles in a DNA Damage Response Pathway
R01 ES 01662512/01/08-11/30/11$2,361
The Children's Oncology Group Chairs Grant
U10 CA 09854303/01/11-02/28/14$12,831
Mechanisms of RET/PTC Rearrangement in Thyroid Cancer
R01 CA 08804103/01/09-02/28/12$6,667
Childhood Cancer Survivor Study
U24 CA 5572712/01/05-11/30/11$60,000
Prediction Model for Radiation Sensitivity in Children with Cancer
U01 CA 13927507/16/09-06/30/11$128,518

Filipovich, A

Gene Therapy for SCID-X1 Using Self-Inactivating (SIN) Gammaretroviral Vector
U01 AI 08762809/01/10-08/31/15$133,693
Histiocyte Society Annual Meeting in Boston
R13 HL 10692502/01/11-01/31/12$5,000
Rare Diseases Clinical Consortia for the Rare Diseases
U54 AI 08297309/12/09-08/31/14$44,978
Hypoxia and Potassium Channel Activity in T Lymphocytes
R01 CA 09528606/01/09-06/30/14$19,959

Jordan, M

Hybrid Immunotherapy for Hemophagocytic Lymphohistiocytosis
01/01/11-12/31/11$47,500

Joshi, S

09-SQOL Study
10/01/10-01/01/12$1,500

Kumar, A

Novel Therapy Development for Infant Leukemia
07/01/10-06/30/11$20,000
Molecular Pathogenesis of MLL-Fusion Gene Leukemia
K08 CA 12219108/19/09-06/30/12$125,250

Marsh, R

Studies to Determine Why XIAP Deficiency Leads to HLH
07/01/10-06/30/12$85,000

Myers, K

Leukemogenesis, Genomic Instability and Fanconi Anemia
07/01/10-06/30/11$20,000

Sumegi, J

Epigenetic Control Of Tumorigenesis In Ewing's Sarcoma Family of Tumors
07/01/10-06/30/11$20,000
Gene-Expression Profiling of Peripheral Blood Mononuclear Cells in Hemophagocytic Lymphohistiocytosis
12/01/10-11/30/11$47,500
Identification of Genes Involved in FHLH
R21 AI 07674609/01/09-08/31/11$125,000
Current Year Direct$917,385
Industry Contracts

Grimley, M

$7,700

Harris, R

$924
Current Year Direct Receipts$8,624
Funded Collaborative Efforts

Bleesing, J

Cincinnati Multidisciplinary Clinical Research Center
Glass, D08/18/08-07/31/133%
Total$926,009
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