Yueh-Yun Chi

Dr. Yueh-Yun Chi is the Director of the Biostatistics Core for the Cancer and Blood Disease Institute (CBDI) at Children’s Hospital Los Angeles (CHLA) and Professor of Research Pediatrics and Population and Public Health Sciences at Keck School of Medicine of USC.

Dr. Chi provides leadership for biostatistical support for faculty, trainees, and staff of the CBDI. Under her leadership, the CBDI Biostatistics Core has doubled in size and capacity in 2022. Dr. Chi also provides statistical oversight for the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) and the New Approaches to Neuroblastoma Therapy (NANT) consortia, in which she has been able to refine and improve study design and conduct for existing and new trials. Prior to joining USC/CHLA, she was a faculty member at the University of Florida and served as the lead statistician for the Renal Tumors and Soft Tissue Committees of the Children’s Oncology Group, the world’s largest research organization dedicated exclusively to pediatric cancer research.

• Clinical characteristics of tumor-induced osteomalacia lesions in the neurocranium and intracranial structures. Orphanet J Rare Dis. 2026 Jul 07.. View in PubMed
• Cost-effectiveness thresholds for initiating osteoporosis treatment in postmenopausal women in China: a microsimulation analysis based on real-world data. Arch Osteoporos. 2026 06 22; 21(1).. View in PubMed
• Ixazomib with chemotherapy for childhood relapsed acute lymphoblastic leukemia: a TACL consortium report. Blood Neoplasia. 2026 Aug; 3(3):100247.. View in PubMed
• A Split sfGFP-Based Signal-Amplified Reporter System for Enhanced Detection of Promoter Activity. Biotechnol J. 2026 May; 21(5):e70244.. View in PubMed
• Elevated calcitonin gene-related peptide expression through prostaglandin E2-EP4 receptor pathway in promoting proliferative periosteum formation in primary hypertrophic osteoarthropathy. J Orthop Translat. 2026 May; 58:101096.. View in PubMed
• Association of serum periostin with bone microarchitecture, muscle mass and function in Chinese postmenopausal women. Front Endocrinol (Lausanne). 2026; 17:1808866.. View in PubMed
• A multi-institutional report of children with atypical teratoid rhabdoid tumor treated with intensive multimodal therapy. Neurooncol Pract. 2026 Aug; 13(4):807-815.. View in PubMed
• Denosumab in pediatric patients with fibrous dysplasia/McCune-Albright syndrome: a single-center, open-labeled study. Front Endocrinol (Lausanne). 2026; 17:1762370.. View in PubMed
• Relationship Between Serum Nell-1 Level and Bone Geometry, Bone Microarchitecture in Chinese Postmenopausal Women. Int J Endocrinol. 2026; 2026:9977862.. View in PubMed
• Association of body fat distribution with bone mineral density: evidence from observational and mendelian randomization analyses. Bone. 2026 03; 204:117742.. View in PubMed
• A nomogram for predicting surgery outcome of tumor-induced osteomalacia: a case-control study. Osteoporos Int. 2025 Dec; 36(12):2567-2577.. View in PubMed
• Evaluation of Quality and Bone Microarchitecture Alterations in Osteopetrosis Patients: Assessed by HR-PQCT. J Clin Endocrinol Metab. 2025 Sep 16; 110(10):e3215-e3224.. View in PubMed
• Glucose Metabolic Abnormalities and Their Interaction With Defective Phosphate Homeostasis in Tumor-induced Osteomalacia. J Clin Endocrinol Metab. 2025 Sep 16; 110(10):2869-2878.. View in PubMed
• Secondary Lung Cancer among Survivors of Adolescent and Young Adult Cancer: A Population-Based Study. Cancer Epidemiol Biomarkers Prev. 2025 Sep 02; 34(9):1618-1626.. View in PubMed
• The genetic polymorphism of XPR1 associated with Fanconi syndrome in Chinese patients with X-linked hypophosphatemia. J Endocrinol Invest. 2025 Dec; 48(12):2869-2878.. View in PubMed
• Generation of human induced pluripotent stem cells with inducible expression of GATA6（Liver cells from hiPSC-GATA6eGFP）. Stem Cell Res. 2025 Sep; 87:103784.. View in PubMed
• Targeting Metabolomics in Primary Hypertrophic Osteoarthropathy: Uncovering Novel Insights into Disease Pathogenesis. J Clin Endocrinol Metab. 2025 Jul 15; 110(8):e2591-e2604.. View in PubMed
• Bone Microarchitecture Evaluated by HR-PQCT in Chinese Adolescent and Pediatric Patients With X-Linked Hypophosphatemia. J Clin Endocrinol Metab. 2025 Jul 15; 110(8):2172-2181.. View in PubMed
• Efficacy and safety of narlumosbart, an anti-RANKL monoclonal antibody, in postmenopausal women with osteoporosis: a multi-center, randomized, double-blind, placebo- and active-controlled, phased II study. EClinicalMedicine. 2025 Jul; 85:103329.. View in PubMed
• Phase I Study of 131I-Metaiodobenzylguanidine With Dinutuximab ± Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial. J Clin Oncol. 2025 08; 43(22):2490-2501.. View in PubMed
• Advancing Protein-Coding RNA Engineering: From Structural Refinement to Biomedical Implementation. Biotechnol J. 2025 Jun; 20(6):e70038.. View in PubMed
• Real-World Experience Using Sodium Thiosulfate Pentahydrate Off-Label for Cisplatin Otoprotection in Children, Adolescents, and Young Adults. Pediatr Blood Cancer. 2025 May; 72(5):e31631.. View in PubMed
• Vincristine Sulfate Liposome Injection With Combination Chemotherapy for Children, Adolescents, and Young Adults With Relapsed Acute Lymphoblastic Leukemia: A Therapeutic Advances in Childhood Leukemia and Lymphoma Consortium Trial. Pediatr Blood Cancer. 2025 May; 72(5):e31584.. View in PubMed
• Optimizing early phase clinical trial washout periods: a report from the Therapeutic Advances in Childhood Leukemia and Lymphoma consortium. J Natl Cancer Inst. 2024 Nov 01; 116(11):1721-1729.. View in PubMed
• A compact, versatile drug-induced splicing switch system with minimal background expression. Cell Rep Methods. 2024 Sep 16; 4(9):100842.. View in PubMed
• Generation of human induced pluripotent stem cells carrying albumin-sfGFP reporter. Stem Cell Res. 2024 12; 81:103543.. View in PubMed
• Clinical characteristics and surgical outcomes of vertebral lesions associated with tumor-induced osteomalacia: report of 16 patients and review of the literature. Osteoporos Int. 2024 Nov; 35(11):1951-1962.. View in PubMed
• A modified glycosylase base editor without predictable DNA off-target effects. FEBS Lett. 2024 Oct; 598(20):2557-2565.. View in PubMed
• Improvement of TaC9-ABE mediated correction of human SMN2 gene. Biotechnol Bioeng. 2024 Oct; 121(10):3059-3067.. View in PubMed
• Response to Letter to the Editor From Judit Tőke and Miklós Tóth: “Shift in Calcium From Peripheral Bone to Axial Bone After Tumor Resection in Patients With Tumor-Induced Osteomalacia”. J Clin Endocrinol Metab. 2024 06 17; 109(7):e1558.. View in PubMed
• Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment. Nat Commun. 2024 Apr 18; 15(1):3258.. View in PubMed
• Nivolumab Plus 5-Azacitidine in Pediatric Relapsed/Refractory Acute Myeloid Leukemia (AML): Phase I/II Trial Results from the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) Consortium. Cancers (Basel). 2024 Jan 24; 16(3).. View in PubMed
• Phase 1 study of high-dose DFMO, celecoxib, cyclophosphamide and topotecan for patients with relapsed neuroblastoma: a New Approaches to Neuroblastoma Therapy trial. Br J Cancer. 2024 03; 130(5):788-797.. View in PubMed
• Stability of African swine fever virus genome under different environmental conditions. Vet World. 2023 Nov; 16(11):2374-2381.. View in PubMed
• Shift in Calcium From Peripheral Bone to Axial Bone After Tumor Resection in Patients With Tumor-Induced Osteomalacia. J Clin Endocrinol Metab. 2023 10 18; 108(11):e1365-e1373.. View in PubMed
• Survival outcomes and surgical morbidity based on surgical approach to pulmonary metastasectomy in pediatric, adolescent and young adult patients with osteosarcoma. Cancer Med. 2023 10; 12(20):20231-20241.. View in PubMed
• Influence of Obesity and Changes in Weight or BMI on Incident Fractures in Postmenopausal Women: From Peking Vertebral Fracture Study. Calcif Tissue Int. 2023 11; 113(5):483-495.. View in PubMed
• Incident vertebral fracture and longitudinal BMD change in Chinese postmenopausal women with early CKD: Peking Vertebral Fracture Study. Arch Osteoporos. 2023 09 11; 18(1):116.. View in PubMed
• Mini-PE, a prime editor with compact Cas9 and truncated reverse transcriptase. Mol Ther Nucleic Acids. 2023 Sep 12; 33:890-897.. View in PubMed
• Improved USER cloning for TALE assembly and its application to base editing. PLoS One. 2023; 18(8):e0289509.. View in PubMed
• Ultra-High Performance Liquid Chromatography-Mass Spectrometry-based Serum Metabolomics for Early Diagnosis of Refractory Tumor-induced Osteomalacia: A Case-control Study. J Clin Endocrinol Metab. 2023 07 14; 108(8):2016-2023.. View in PubMed
• Intravenous N-Acetylcysteine to Prevent Cisplatin-Induced Hearing Loss in Children: A Nonrandomized Controlled Phase I Trial. Clin Cancer Res. 2023 07 05; 29(13):2410-2418.. View in PubMed
• Generation of hiPSCs with ABO c.767T>C substitution: resulting in splicing variants. Front Genet. 2023; 14:1141756.. View in PubMed
• Generation of a homozygous RANGRF knockout hiPSC line by CRISPR/Cas9 system. Stem Cell Res. 2023 09; 71:103136.. View in PubMed
• Comparison of bone microstructure and strength in the distal radius and tibia between the different types of primary hypertrophic osteoarthropathy: an HR-pQCT study. Osteoporos Int. 2023 Aug; 34(8):1453-1464.. View in PubMed
• TP53 germline pathogenic variant frequency in anaplastic rhabdomyosarcoma: A Children’s Oncology Group report. Pediatr Blood Cancer. 2023 May 17.. View in PubMed
• Generation of a homozygous GRIN2A gene knockout human embryonic stem cell line using CRISPR/Cas9 system. Stem Cell Res. 2023 06; 69:103121.. View in PubMed
• Relationships between sclerostin and morphometric vertebral fractures, bone mineral density, and bone microarchitecture in postmenopausal women. Arch Osteoporos. 2023 04 29; 18(1):57.. View in PubMed
• Hyperparathyroidism in a Large Cohort of Chinese Patients With Tumor-induced Osteomalacia. J Clin Endocrinol Metab. 2023 04 13; 108(5):1224-1235.. View in PubMed
• Alveolar rhabdomyosarcoma has superior response rates to vinorelbine compared to embryonal rhabdomyosarcoma in patients with relapsed/refractory disease: A meta-analysis. Cancer Med. 2023 05; 12(9):10222-10229.. View in PubMed
• Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results. Nat Med. 2023 05; 29(5):1092-1102.. View in PubMed
• The First Compound Heterozygous Mutations of DMP1 Causing Rare Autosomal Recessive Hypophosphatemic Rickets Type 1. J Clin Endocrinol Metab. 2023 03 10; 108(4):791-801.. View in PubMed
• Serum Irisin level is associated with fall risk, muscle strength, and cortical porosity in postmenopausal women. Front Endocrinol (Lausanne). 2023; 14:1096950.. View in PubMed
• Bcl-xL Promotes the Survival of Motor Neurons Derived from Neural Stem Cells. Biology (Basel). 2023 Jan 13; 12(1).. View in PubMed
• Robust genetic model-based SNP-set association test using CauchyGM. Bioinformatics. 2023 01 01; 39(1).. View in PubMed
• Efficient and Safe Editing of Porcine Endogenous Retrovirus Genomes by Multiple-Site Base-Editing Editor. Cells. 2022 12 08; 11(24).. View in PubMed
• Clinical characteristics and the influence of rs1800470 in patients with Camurati-Engelmann disease. Front Endocrinol (Lausanne). 2022; 13:1041061.. View in PubMed
• Temsirolimus combined with cyclophosphamide and etoposide for pediatric patients with relapsed/refractory acute lymphoblastic leukemia: a Therapeutic Advances in Childhood Leukemia Consortium trial (TACL 2014-001). Haematologica. 2022 10 01; 107(10):2295-2303.. View in PubMed
• Tumor-induced osteomalacia combined with acromegaly: A case report. Ann N Y Acad Sci. 2022 11; 1517(1):88-92.. View in PubMed
• Na-Cl Co-transporter (NCC) gene inactivation is associated with improved bone microstructure. Osteoporos Int. 2022 Oct; 33(10):2193-2204.. View in PubMed
• Bone Volumetric Density, Microarchitecture, and Estimated Bone Strength in Tumor-Induced Rickets/Osteomalacia Versus X-linked Hypophosphatemia in Chinese Adolescents. Front Endocrinol (Lausanne). 2022; 13:883981.. View in PubMed
• ASO Video Abstract: Kidney Preservation and Wilms Tumor Development in Children with Diffuse Hyperplastic Perilobar Nephroblastomatosis-A Report from the Children’s Oncology Group Study AREN0534. Ann Surg Oncol. 2022 05; 29(5):3262-3263.. View in PubMed
• Outcomes based on histopathologic response to preoperative chemotherapy in children with bilateral Wilms tumor: A prospective study (COG AREN0534). Cancer. 2022 07 01; 128(13):2493-2503.. View in PubMed
• Decitabine and vorinostat with FLAG chemotherapy in pediatric relapsed/refractory AML: Report from the therapeutic advances in childhood leukemia and lymphoma (TACL) consortium. Am J Hematol. 2022 05; 97(5):613-622.. View in PubMed
• Survival of pediatric Hodgkin lymphoma patients treated with doxorubicin, bleomycin, vincristine, etoposide, prednisone, and cyclophosphamide (ABVE-PC) versus doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD) at a single institution. Pediatr Blood Cancer. 2022 05; 69(5):e29601.. View in PubMed
• Independent Assessment of the Children’s Hepatic Tumors International Collaboration Risk Stratification for Hepatoblastoma and the Association of Tumor Histological Characteristics With Prognosis. JAMA Netw Open. 2022 02 01; 5(2):e2148013.. View in PubMed
• Kidney Preservation and Wilms Tumor Development in Children with Diffuse Hyperplastic Perilobar Nephroblastomatosis: A Report from the Children’s Oncology Group Study AREN0534. Ann Surg Oncol. 2022 May; 29(5):3252-3261.. View in PubMed
• Bone Impairment in a Large Cohort of Chinese Patients With Tumor-Induced Osteomalacia Assessed by HR-pQCT and TBS. J Bone Miner Res. 2022 03; 37(3):454-464.. View in PubMed
• Rising drug cost impacts on cost-effectiveness of 2 chemotherapy regimens for intermediate-risk rhabdomyosarcoma: A report from the Children’s Oncology Group. Cancer. 2022 Jan 15; 128(2):317-325.. View in PubMed
• Ultra-High Dose Vitamin D in Pediatric Hematopoietic Stem Cell Transplantation: A Nonrandomized Controlled Trial. Transplant Cell Ther. 2021 12; 27(12):1001.e1-1001.e9.. View in PubMed
• Relationships between reproductive hormones and maternal pregnancy physiology in women conceiving with or without in vitro fertilization. Am J Physiol Regul Integr Comp Physiol. 2021 09 01; 321(3):R454-R468.. View in PubMed
• Haplotype-resolved germline and somatic alterations in renal medullary carcinomas. Genome Med. 2021 07 14; 13(1):114.. View in PubMed
• Germline Cancer Predisposition Variants in Pediatric Rhabdomyosarcoma: A Report From the Children’s Oncology Group. J Natl Cancer Inst. 2021 07 01; 113(7):875-883.. View in PubMed
• Early Discrimination Between Tumor-Induced Rickets/Osteomalacia and X-Linked Hypophosphatemia in Chinese Children and Adolescents: A Retrospective Case-Control Study. J Bone Miner Res. 2021 09; 36(9):1739-1748.. View in PubMed
• Consensus on clinical management of tumor-induced osteomalacia. Chin Med J (Engl). 2021 Apr 01; 134(11):1264-1266.. View in PubMed
• Local Control For High-Grade Nonrhabdomyosarcoma Soft Tissue Sarcoma Assigned to Radiation Therapy on ARST0332: A Report From the Childrens Oncology Group. Int J Radiat Oncol Biol Phys. 2021 Jul 01; 110(3):821-830.. View in PubMed
• Metabolic response as assessed by 18 F-fluorodeoxyglucose positron emission tomography-computed tomography does not predict outcome in patients with intermediate- or high-risk rhabdomyosarcoma: A report from the Children’s Oncology Group Soft Tissue Sarcoma Committee. Cancer Med. 2021 02; 10(3):857-866.. View in PubMed
• The prognostic significance of anaplasia in childhood rhabdomyosarcoma: A report from the Children’s Oncology Group. Eur J Cancer. 2021 01; 143:127-133.. View in PubMed
• Alveolar rhabdomyosarcoma with regional nodal involvement: Results of a combined analysis from two cooperative groups. Pediatr Blood Cancer. 2021 03; 68(3):e28832.. View in PubMed
• Survival outcomes of patients with localized FOXO1 fusion-positive rhabdomyosarcoma treated on recent clinical trials: A report from the Soft Tissue Sarcoma Committee of the Children’s Oncology Group. Cancer. 2021 03 15; 127(6):946-956.. View in PubMed
• Benefit of delayed primary excision in rhabdomyosarcoma: A report from the Children’s Oncology Group. Cancer. 2021 01 15; 127(2):275-283.. View in PubMed
• A prospective study of pediatric and adolescent renal cell carcinoma: A report from the Children’s Oncology Group AREN0321 study. Cancer. 2020 12 01; 126(23):5156-5164.. View in PubMed
• Pathological response in children and adults with large unresected intermediate-grade or high-grade soft tissue sarcoma receiving preoperative chemoradiotherapy with or without pazopanib (ARST1321): a multicentre, randomised, open-label, phase 2 trial. Lancet Oncol. 2020 08; 21(8):1110-1122.. View in PubMed
• Impact of local control and surgical lymph node evaluation in localized paratesticular rhabdomyosarcoma: A report from the Children’s Oncology Group Soft Tissue Sarcoma Committee. Int J Cancer. 2020 12 01; 147(11):3168-3176.. View in PubMed
• Results of Treatment for Patients With Multicentric or Bilaterally Predisposed Unilateral Wilms Tumor (AREN0534): A report from the Children’s Oncology Group. Cancer. 2020 08 01; 126(15):3516-3525.. View in PubMed
• Maternal endothelial function, circulating endothelial cells, and endothelial progenitor cells in pregnancies conceived with or without in vitro fertilization. Am J Physiol Regul Integr Comp Physiol. 2020 06 01; 318(6):R1091-R1102.. View in PubMed
• Outcome analysis of stage I epithelial-predominant favorable-histology Wilms tumors: A report from Children’s Oncology Group study AREN03B2. Cancer. 2020 06 15; 126(12):2866-2871.. View in PubMed
• Imaging Characteristics of Nephrogenic Rests Versus Small Wilms Tumors: A Report From the Children’s Oncology Group Study AREN03B2. AJR Am J Roentgenol. 2020 05; 214(5):987-994.. View in PubMed
• Activity of Vincristine and Irinotecan in Diffuse Anaplastic Wilms Tumor and Therapy Outcomes of Stage II to IV Disease: Results of the Children’s Oncology Group AREN0321 Study. J Clin Oncol. 2020 05 10; 38(14):1558-1568.. View in PubMed
• Do children and adolescents with completely resected alveolar rhabdomyosarcoma require adjuvant radiation? A report from the Children’s Oncology Group. Pediatr Blood Cancer. 2020 05; 67(5):e28243.. View in PubMed
• Relationship between tumor response at therapy completion and prognosis in patients with Group III rhabdomyosarcoma: A report from the Children’s Oncology Group. Int J Cancer. 2020 09 01; 147(5):1419-1426.. View in PubMed
• Reply to D.M. Green. J Clin Oncol. 2020 03 01; 38(7):773-774.. View in PubMed
• An efficient integrative resampling method for gene-trait association analysis. Genet Epidemiol. 2020 03; 44(2):197-207.. View in PubMed
• A risk-based treatment strategy for non-rhabdomyosarcoma soft-tissue sarcomas in patients younger than 30 years (ARST0332): a Children’s Oncology Group prospective study. Lancet Oncol. 2020 01; 21(1):145-161.. View in PubMed
• Nonremission and Recurrent Tumor-Induced Osteomalacia: A Retrospective Study. J Bone Miner Res. 2020 03; 35(3):469-477.. View in PubMed
• Randomized Phase II Trial of Bevacizumab or Temsirolimus in Combination With Chemotherapy for First Relapse Rhabdomyosarcoma: A Report From the Children’s Oncology Group. J Clin Oncol. 2019 11 01; 37(31):2866-2874.. View in PubMed
• Refinement of risk stratification for childhood rhabdomyosarcoma using FOXO1 fusion status in addition to established clinical outcome predictors: A report from the Children’s Oncology Group. Cancer Med. 2019 10; 8(14):6437-6448.. View in PubMed
• Augmentation of Therapy for Combined Loss of Heterozygosity 1p and 16q in Favorable Histology Wilms Tumor: A Children’s Oncology Group AREN0532 and AREN0533 Study Report. J Clin Oncol. 2019 10 20; 37(30):2769-2777.. View in PubMed
• Potential influence of the corpus luteum on circulating reproductive and volume regulatory hormones, angiogenic and immunoregulatory factors in pregnant women. Am J Physiol Endocrinol Metab. 2019 10 01; 317(4):E677-E685.. View in PubMed
• Prohibitin is a prognostic marker and therapeutic target to block chemotherapy resistance in Wilms’ tumor. JCI Insight. 2019 08 08; 4(15).. View in PubMed

• Gill, Inderbir
• Lenz, Heinz-Josef
• Matsuo, Koji
• Daneshmand, Siamak
• Renfro, Lindsay
• Demetriades, Demetrios