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4. Bone mineral density surveillance for childhood, adolescent, and young adult cancer survivors: evidence-based recommendations from the International Late Effects of Childhood Cancer Guideline Harmonization Group

Author

van Atteveld, Jenneke E, Mulder, Renée L, van den Heuvel-Eibrink, Marry M, Hudson, Melissa M, Kremer, Leontien C M, Skinner, Roderick, Wallace, W Hamish, Constine, Louis S, Higham, Claire E, Kaste, Sue C, Niinimäki, Riitta, Mostoufi-Moab, Sogol, Alos, Nathalie, Fintini, Danilo, Templeton, Kimberly J, Ward, Leanne M, Frey, Eva, Franceschi, Roberto, Pavasovic, Vesna, Karol, Seth E, Amin, Nadia L, Vrooman, Lynda M, Harila-Saari, Arja, Demoor-Goldschmidt, Charlotte, Murray, Robert D, Bardi, Edit, Lequin, Maarten H, Faienza, Maria Felicia, Zaikova, Olga, Berger, Claire, Mora, Stefano, Ness, Kirsten K, Neggers, Sebastian J C M M, Pluijm, Saskia M F, Simmons, Jill H, and Di Iorgi, Natascia

Published
2021
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10. Quantitative computed tomography analysis of body composition changes in paediatric patients with acute lymphoblastic leukaemia

Author

Suwannaying, Kunanya, primary, Ong, Adrian A., additional, Dhaduk, Rikeenkumar, additional, Pei, Deqing, additional, Iijima, Mayuko, additional, Merkle, Eric, additional, Zhuang, Tony Z., additional, Goodenough, Chelsea G., additional, Brown, Joren, additional, Browne, Emily K., additional, Wolcott, Bruce, additional, Cheng, Cheng, additional, Wilson, Carmen L., additional, Pui, Ching‐Hon, additional, Ness, Kirsten K., additional, Kaste, Sue C., additional, and Inaba, Hiroto, additional

Published
2024
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12. Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog–Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032

Author

Robinson, Giles W, Orr, Brent A, Wu, Gang, Gururangan, Sridharan, Lin, Tong, Qaddoumi, Ibrahim, Packer, Roger J, Goldman, Stewart, Prados, Michael D, Desjardins, Annick, Chintagumpala, Murali, Takebe, Naoko, Kaste, Sue C, Rusch, Michael, Allen, Sariah J, Onar-Thomas, Arzu, Stewart, Clinton F, Fouladi, Maryam, Boyett, James M, Gilbertson, Richard J, Curran, Tom, Ellison, David W, and Gajjar, Amar

Subjects
Rare Diseases, Neurosciences, Pediatric Research Initiative, Brain Cancer, Cancer, Clinical Research, Pediatric, Brain Disorders, Pediatric Cancer, Genetics, Human Genome, Adult, Anilides, Brain Neoplasms, Female, Hedgehog Proteins, Humans, Male, Medulloblastoma, Middle Aged, Pyridines, Young Adult, Clinical Sciences, Oncology and Carcinogenesis, Oncology & Carcinogenesis
Abstract

PurposeTwo phase II studies assessed the efficacy of vismodegib, a sonic hedgehog (SHH) pathway inhibitor that binds smoothened (SMO), in pediatric and adult recurrent medulloblastoma (MB).Patients and methodsAdult patients enrolled onto PBTC-025B and pediatric patients enrolled onto PBTC-032 were treated with vismodegib (150 to 300 mg/d). Protocol-defined response, which had to be sustained for 8 weeks, was confirmed by central neuroimaging review. Molecular tests to identify patterns of response and insensitivity were performed when tissue was available.ResultsA total of 31 patients were enrolled onto PBTC-025B, and 12 were enrolled onto PBTC-032. Three patients in PBTC-025B and one in PBTC-032, all with SHH-subgroup MB (SHH-MB), exhibited protocol-defined responses. Progression-free survival (PFS) was longer in those with SHH-MB than in those with non-SHH-MB, and prolonged disease stabilization occurred in 41% of patient cases of SHH-MB. Among those with SHH-MB, loss of heterozygosity of PTCH1 was associated with prolonged PFS, and diffuse staining of P53 was associated with reduced PFS. Whole-exome sequencing identified mutations in SHH genes downstream from SMO in four of four tissue samples from nonresponders and upstream of SMO in two of four patients with favorable responses.ConclusionVismodegib exhibits activity against adult recurrent SHH-MB but not against recurrent non-SHH-MB. Inadequate accrual of pediatric patients precluded conclusions in this population. Molecular analyses support the hypothesis that SMO inhibitor activity depends on the genomic aberrations within the tumor. Such inhibitors should be advanced in SHH-MB studies; however, molecular and genomic work remains imperative to identify target populations that will truly benefit.

Published
2015

17. Long-Term Functional Outcomes Among Childhood Survivors of Cancer Who Have a History of Osteonecrosis

Author

DeFeo, Brian M., Kaste, Sue C., Li, Zhenghong, Brinkman, Tara M., Neel, Michael D., Srivastava, Deo Kumar, Hudson, Melissa M., Robison, Leslie L., Karoi, Seth E., and Ness, Kirsten K.

Subjects
Oncology, Experimental, Bones -- Necrosis, Corticosteroids -- Research, Non-Hodgkin's lymphomas -- Research -- Patient outcomes, Cancer -- Patient outcomes -- Research, Health
Abstract

Background. Glucocorticoids used to treat childhood leukemia and lymphoma can result in osteonecrosis, leading to physical dysfunction and pain. Improving survival rates warrants research into long-term outcomes among this population. Objective. The objective of this study was to compare the physical function and quality of life (QOL) of survivors of childhood cancer who had an osteonecrosis history with that of survivors who had no osteonecrosis history and with that of people who were healthy (controls). Design. This was a cross-sectional study. Methods. This study included St Jude Lifetime Cohort Study participants who were [greater than or equal to] 10 years from the diagnosis of childhood leukemia or lymphoma and [greater than or equal to] 18 years old; 135 had osteonecrosis (52.5% men; mean age = 27.7 [SD = 6.08] years) and 1560 had no osteonecrosis history (52.4% men; mean age = 33.3 [SD = 8.54] years). This study also included 272 people who were from the community and who were healthy (community controls) (47.7% men; mean age = 35.1 [SD = 10.46] years). The participants completed functional assessments and questionnaires about QOL. Results. Survivors with osteonecrosis scored lower than other survivors and controls for dorsiflexion strength (mean score = 16.50 [SD = 7.91] vs 24.17 [SD = 8.61] N*m/kg) and scored lower than controls for flexibility with the sit-and-reach test (20.61 [SD = 9.70] vs 23.96 [SD = 10.73] cm), function on the Physical Performance Test (mean score = 22.73 [SD = 2.05] vs 23.58 [SD = 0.88]), and mobility on the Timed 'Up & Go' Test (5.66 [SD = 2.25] vs 5.12 [SD = 1.28] seconds). Survivors with hip osteonecrosis requiring surgery scored lower than survivors without osteonecrosis for dorsiflexion strength (13.75 [SD = 8.82] vs 18.48 [SD = 9.04] Nm/kg), flexibility (15.79 [SD = 8.93] vs 20.37 [SD = 10.14] cm), and endurance on the 6-minute walk test (523.50 [SD = 103.00] vs 572.10 [SD = 102.40] m). Limitations. Because some eligible survivors declined to participate, possible selection bias was a limitation of this study. Conclusions. survivors of childhood leukemia and lymphoma with and without osteonecrosis demonstrated impaired physical performance and reported reduced QOL compared with controls, with those requiring surgery for osteonecrosis most at risk for impairments. It may be beneficial to provide strengthening, flexibility, and endurance interventions for patients who have pediatric cancer and osteonecrosis for long-term function., Survival rates continue to improve among children treated for leukemia and lymphoma such that the vast majority of these children will survive their disease. (1) Five-year relative survival rates for [...]

Published
2020
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20. Soft Tissue Sarcomas

Author

Parham, David M., Kaste, Sue C., Raju, Anand, McCarville, M. Beth, Parham, David M., editor, Khoury, Joseph D., editor, and McCarville, M. Beth, editor

Published
2015
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22. Feasibility of indocyanine green‐guided localization of pulmonary nodules in children with solid tumors

Author

Abdelhafeez, Abdelhafeez H., primary, Mothi, Suraj Sarvode, additional, Pio, Luca, additional, Mori, Motomi, additional, Santiago, Teresa C., additional, McCarville, M. Beth, additional, Kaste, Sue C., additional, Pappo, Alberto S., additional, Talbot, Lindsay J., additional, Murphy, Andrew J., additional, and Davidoff, Andrew M., additional

Published
2023
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23. Data from Approach for Classification and Severity Grading of Long-term and Late-Onset Health Events among Childhood Cancer Survivors in the St. Jude Lifetime Cohort

Author

Hudson, Melissa M., primary, Ehrhardt, Matthew J., primary, Bhakta, Nickhill, primary, Baassiri, Malek, primary, Eissa, Hesham, primary, Chemaitilly, Wassim, primary, Green, Daniel M., primary, Mulrooney, Daniel A., primary, Armstrong, Gregory T., primary, Brinkman, Tara M., primary, Klosky, James L., primary, Krull, Kevin R., primary, Sabin, Noah D., primary, Wilson, Carmen L., primary, Huang, I-Chan, primary, Bass, Johnnie K., primary, Hale, Karen, primary, Kaste, Sue, primary, Khan, Raja B., primary, Srivastava, Deo Kumar, primary, Yasui, Yutaka, primary, Joshi, Vijaya M., primary, Srinivasan, Saumini, primary, Stokes, Dennis, primary, Hoehn, Mary Ellen, primary, Wilson, Matthew, primary, Ness, Kirsten K., primary, and Robison, Leslie L., primary

Published
2023
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24. Supplemental Table 2 from Approach for Classification and Severity Grading of Long-term and Late-Onset Health Events among Childhood Cancer Survivors in the St. Jude Lifetime Cohort

Author

Hudson, Melissa M., primary, Ehrhardt, Matthew J., primary, Bhakta, Nickhill, primary, Baassiri, Malek, primary, Eissa, Hesham, primary, Chemaitilly, Wassim, primary, Green, Daniel M., primary, Mulrooney, Daniel A., primary, Armstrong, Gregory T., primary, Brinkman, Tara M., primary, Klosky, James L., primary, Krull, Kevin R., primary, Sabin, Noah D., primary, Wilson, Carmen L., primary, Huang, I-Chan, primary, Bass, Johnnie K., primary, Hale, Karen, primary, Kaste, Sue, primary, Khan, Raja B., primary, Srivastava, Deo Kumar, primary, Yasui, Yutaka, primary, Joshi, Vijaya M., primary, Srinivasan, Saumini, primary, Stokes, Dennis, primary, Hoehn, Mary Ellen, primary, Wilson, Matthew, primary, Ness, Kirsten K., primary, and Robison, Leslie L., primary

Published
2023
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25. Supplementary Tables 1 - 3 from Phase I Study of Vismodegib in Children with Recurrent or Refractory Medulloblastoma: A Pediatric Brain Tumor Consortium Study

Author

Gajjar, Amar, primary, Stewart, Clinton F., primary, Ellison, David W., primary, Kaste, Sue, primary, Kun, Larry E., primary, Packer, Roger J., primary, Goldman, Stewart, primary, Chintagumpala, Murali, primary, Wallace, Dana, primary, Takebe, Naoko, primary, Boyett, James M., primary, Gilbertson, Richard J., primary, and Curran, Tom, primary

Published
2023
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26. Supplementary Methods from Phase I Trial, Pharmacokinetics, and Pharmacodynamics of Vandetanib and Dasatinib in Children with Newly Diagnosed Diffuse Intrinsic Pontine Glioma

Author

Broniscer, Alberto, primary, Baker, Sharyn D., primary, Wetmore, Cynthia, primary, Pai Panandiker, Atmaram S., primary, Huang, Jie, primary, Davidoff, Andrew M., primary, Onar-Thomas, Arzu, primary, Panetta, John C., primary, Chin, Thomas K., primary, Merchant, Thomas E., primary, Baker, Justin N., primary, Kaste, Sue C., primary, Gajjar, Amar, primary, and Stewart, Clinton F., primary

Published
2023
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27. Supplementary Figures 1 - 2 from Phase I Study of Vismodegib in Children with Recurrent or Refractory Medulloblastoma: A Pediatric Brain Tumor Consortium Study

Author

Gajjar, Amar, primary, Stewart, Clinton F., primary, Ellison, David W., primary, Kaste, Sue, primary, Kun, Larry E., primary, Packer, Roger J., primary, Goldman, Stewart, primary, Chintagumpala, Murali, primary, Wallace, Dana, primary, Takebe, Naoko, primary, Boyett, James M., primary, Gilbertson, Richard J., primary, and Curran, Tom, primary

Published
2023
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28. Supplemental Table 1 from Approach for Classification and Severity Grading of Long-term and Late-Onset Health Events among Childhood Cancer Survivors in the St. Jude Lifetime Cohort

Author

Hudson, Melissa M., primary, Ehrhardt, Matthew J., primary, Bhakta, Nickhill, primary, Baassiri, Malek, primary, Eissa, Hesham, primary, Chemaitilly, Wassim, primary, Green, Daniel M., primary, Mulrooney, Daniel A., primary, Armstrong, Gregory T., primary, Brinkman, Tara M., primary, Klosky, James L., primary, Krull, Kevin R., primary, Sabin, Noah D., primary, Wilson, Carmen L., primary, Huang, I-Chan, primary, Bass, Johnnie K., primary, Hale, Karen, primary, Kaste, Sue, primary, Khan, Raja B., primary, Srivastava, Deo Kumar, primary, Yasui, Yutaka, primary, Joshi, Vijaya M., primary, Srinivasan, Saumini, primary, Stokes, Dennis, primary, Hoehn, Mary Ellen, primary, Wilson, Matthew, primary, Ness, Kirsten K., primary, and Robison, Leslie L., primary

Published
2023
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31. Genetics of glucocorticoid-associated osteonecrosis in children with acute lymphoblastic leukemia

Author

Karol, Seth E., Yang, Wenjian, Van Driest, Sara L., Chang, Tamara Y., Kaste, Sue, Bowton, Erica, Basford, Melissa, Bastarache, Lisa, Roden, Dan M., Denny, Joshua C., Larsen, Eric, Winick, Naomi, Carroll, William L., Cheng, Cheng, Pei, Deqing, Fernandez, Christian A., Liu, Chengcheng, Smith, Colton, Loh, Mignon L., Raetz, Elizabeth A., Hunger, Stephen P., Scheet, Paul, Jeha, Sima, Pui, Ching-Hon, Evans, William E., Devidas, Meenakshi, Mattano, Leonard A., Jr, and Relling, Mary V.

Published
2015
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38. A Phase IV Trial of Proton Therapy in Children: The First Report from the SJPROTON1 Study

Author

Lucas, John T., primary, Marker, Matthew, additional, Becksfort, Jared, additional, Patni, Tushar, additional, Faught, Austin M., additional, Tinkle, Christopher L., additional, Groben, Valerie J., additional, Burghen, Elizabeth, additional, Ruleman, Haley, additional, Hua, Chia-Ho, additional, Kaste, Sue C., additional, Acharya, Sahaja, additional, Sabin, Noah D., additional, Hudson, Melissa, additional, Wu, Shengjie, additional, Li, Yimei, additional, Krasin, Matthew J., additional, and Merchant, Thomas E., additional

Published
2022
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39. Energy cost of walking in obese survivors of acute lymphoblastic leukemia: A report from the St. Jude Lifetime Cohort

Author

Wogksch, Matthew D., primary, Finch, Emily R., additional, Nolan, Vikki G., additional, Smeltzer, Matthew P., additional, Mzayek, Fawaz, additional, Goodenough, Chelsea G., additional, Pui, Ching-Hon, additional, Inaba, Hiroto, additional, Mulrooney, Daniel A., additional, Kaste, Sue C., additional, Brinkman, Tara M., additional, Lanctot, Jennifer Q., additional, Srivastava, Deo Kumar, additional, Jefferies, John L., additional, Armstrong, Gregory T., additional, Robison, Leslie L., additional, Hudson, Melissa M., additional, and Ness, Kirsten K., additional

Published
2022
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45. Reductions in Bone Mineral Density Are Apparent Early in Children With Prevalent Osteonecrosis Lesions Following Leukemia Therapy.

Author

Halton, Jacqueline M., Ma, Jinui, Babyn, Paul, Matzinger, Mary Ann, Kaste, Sue C., Scharke, Maya, Fernandez, Conrad V., Miettunen, Paivi, Ho, Josephine, Alos, Nathalie, Abish, Sharon, Barr, Ronald, Cairney, Elizabeth, Dix, David B., Grant, Ronald M., Israels, Sara, Lewis, Victor, Wilson, Beverly, Atkinson, Stephanie, and Cabral, David

Abstract

Osteonecrosis (ON) is a serious complication of childhood acute lymphoblastic leukemia. We determined the prevalence of osteonecrotic lesions in our patient population by a one‐time multisite magnetic resonance imaging (MRI) more than 1 year following leukemia therapy. MRI findings were evaluated in relationship to clinical factors (including longitudinal changes in bone mineral density [BMD]). Eighty‐six children enrolled in the Steroid Associated Osteoporosis in the Pediatric Population (STOPP) study were evaluated for ON at 3.1 ± 1.3 years following therapy. Thirty children had a total of 150 confirmed ON lesions (35%). Lumbar spine (LS) BMD Z‐scores (mean ± SD) were low at diagnosis and similar between patients with and without ON (−1.09 ± 1.53 versus −1.27 ± 1.25, p = 0.549). LS BMD Z‐scores declined from baseline to 12 months in children with ON (−0.31 ± 1.02) but not in those without (0.13 ± 0.82, p = 0.035); the hip BMD Z‐scores from baseline to 24 months declined in both groups, but to a greater extent in those with ON (−1.77 ± 1.22) compared to those without (−1.03 ± 1.07, p = 0.045). At the time of the MRI, mean total hip and total body (TB) BMD Z‐scores were lower in children with ON (hip −0.98 ± 0.95 versus −0.28 ± 1.06, p = 0.010; TB −1.36 ± 1.10 versus −0.48 ± 1.50, p = 0.018). Pain occurred in 11/30 (37%) with ON versus 20/56 (36%) without, p = 0.841. In multivariable models, older age at diagnosis (odds ratio [OR] 1.57; 95% confidence interval [CI], 1.15–2.13; p = 0.004), and hip BMD Z‐score at MRI (OR 2.23; 95% CI, 1.02–4.87; p = 0.046) were independently associated with ON. Overall, one‐third of children demonstrated ON after leukemia therapy. Those with ON had greater reductions in spine and hip BMD Z‐scores in the first 1 and 2 years of therapy, respectively. Older age and lower hip BMD Z‐scores at MRI were significantly associated with prevalent, off‐therapy ON. These data assist in identifying children at risk of ON. © 2023 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR). [ABSTRACT FROM AUTHOR]

Published
2023
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50. Evidence‐based surgical guidelines for treating children with Wilms tumor in low‐resource settings

Author

Abdelhafeez, Abdelhafeez H., primary, Reljic, Tea, additional, Kumar, Ambuj, additional, Banu, Tahmina, additional, Cox, Sharon, additional, Davidoff, Andrew M., additional, Elgendy, Ahmed, additional, Ghandour, Khalil, additional, Gerstle, J. Ted, additional, Karpelowsky, Jonathan, additional, Kaste, Sue C., additional, Kechiche, Nahla, additional, Esiashvili, Natia, additional, Nasir, Abdulrasheed, additional, Ngongola, Amon, additional, Marollano, Jin, additional, Moreno, Amabelle A., additional, Muzira, Arlene, additional, Parkes, Jeannette, additional, Saldaña, Lily J., additional, Shalkow, Jaime, additional, Vujanić, Gordan M., additional, Velasquez, Thelma, additional, Lakhoo, Kokila, additional, Mukkada, Sheena, additional, and Abib, Simone, additional

Published
2022
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