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1. Trends in volumes and survival after hematopoietic cell transplantation in racial/ethnic minorities

Author

Khera, Nandita, Ailawadhi, Sikander, Brazauskas, Ruta, Patel, Jinalben, Jacobs, Benjamin, Ustun, Celalettin, Ballen, Karen, Abid, Muhammad Bilal, Diaz Perez, Miguel Angel, Al-Homsi, A. Samer, Hashem, Hasan, Hong, Sanghee, Munker, Reinhold, Schears, Raquel M., Lazarus, Hillard M., Ciurea, Stefan, Badawy, Sherif M., Savani, Bipin N., Wirk, Baldeep, LeMaistre, C. Fred, Bhatt, Neel S., Beitinjaneh, Amer, Aljurf, Mahmoud, Sharma, Akshay, Cerny, Jan, Knight, Jennifer M., Kelkar, Amar H., Yared, Jean A., Kindwall-Keller, Tamila, Winestone, Lena E., Steinberg, Amir, Arnold, Staci D., Seo, Sachiko, Preussler, Jaime M., Hossain, Nasheed M., Fingrut, Warren B., Agrawal, Vaibhav, Hashmi, Shahrukh, Lehmann, Leslie E., Wood, William A., Rangarajan, Hemalatha G., Saber, Wael, and Hahn, Theresa

Published
2024
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2. Comprehensive Prognostication in Critically Ill Pediatric Hematopoietic Cell Transplant Patients: Results from Merging the Center for International Blood and Marrow Transplant Research (CIBMTR) and Virtual Pediatric Systems (VPS) Registries

Author

Zinter, Matt S, Logan, Brent R, Fretham, Caitrin, Sapru, Anil, Abraham, Allistair, Aljurf, Mahmoud D, Arnold, Staci D, Artz, Andrew, Auletta, Jeffery J, Chhabra, Saurabh, Copelan, Edward, Duncan, Christine, Gale, Robert P, Guinan, Eva, Hematti, Peiman, Keating, Amy K, Marks, David I, Olsson, Richard, Savani, Bipin N, Ustun, Celalettin, Williams, Kirsten M, Pasquini, Marcelo C, and Dvorak, Christopher C

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Sciences, Patient Safety, Clinical Research, Pediatric, Rare Diseases, Cancer, Hematology, Transplantation, Good Health and Well Being, Child, Critical Illness, Hematopoietic Stem Cell Transplantation, Humans, Infant, Intensive Care Units, Pediatric, Registries, Retrospective Studies, Risk Factors, Intensive care units, Hematopoietic stem cell transplantation, Organ dysfunction scores, Prognosis, Survival analysis, Immunology, Cardiovascular medicine and haematology
Abstract

Critically ill pediatric allogeneic hematopoietic cell transplant (HCT) patients may benefit from early and aggressive interventions aimed at reversing the progression of multiorgan dysfunction. Therefore, we evaluated 25 early risk factors for pediatric intensive care unit (PICU) mortality to improve mortality prognostication. We merged the Virtual Pediatric Systems and Center for International Blood and Marrow Transplant Research databases and analyzed 936 critically ill patients ≤21 years of age who had undergone allogeneic HCT and subsequently required PICU admission between January 1, 2009, and December 31, 2014. Of 1532 PICU admissions, the overall PICU mortality rate was 17.4% (95% confidence interval [CI], 15.6% to 19.4%) but was significantly higher for patients requiring mechanical ventilation (44.0%), renal replacement therapy (56.1%), or extracorporeal life support (77.8%). Mortality estimates increased significantly the longer that patients remained in the PICU. Of 25 HCT- and PICU-specific characteristics available at or near the time of PICU admission, moderate/severe pre-HCT renal injury, pre-HCT recipient cytomegalovirus seropositivity,

Published
2020

3. Reproductive Health Assessment and Reports of Fertility Counseling in Pediatric and Adolescent Patients with Sickle Cell Disease After Hematopoietic Cell Transplantation

Author

George, Sobenna A., Veludhandi, Anirudh, Xiang, Yijin, Liu, Katie, Stenger, Elizabeth, Arnold, Staci D., Mehta, Akanksha, Schirmer, David A., Spencer, Jessica B., Guilcher, Gregory M.T., Bhatia, Monica, Abraham, Allistair, Gomez-Lobo, Veronica, Krishnamurti, Lakshmanan, and Meacham, Lillian R.

Published
2024
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8. Racial and Socioeconomic Disparities in Long-Term Outcomes in ≥1 Year Allogeneic Hematopoietic Cell Transplantation Survivors: A CIBMTR Analysis

Author

Blue, Brandon J., Brazauskas, Ruta, Chen, Karen, Patel, Jinalben, Zeidan, Amer M., Steinberg, Amir, Ballen, Karen, Kwok, Janette, Rotz, Seth J., Perez, Miguel Angel Diaz, Kelkar, Amar H., Ganguly, Siddhartha, Wingard, John R., Lad, Deepesh, Sharma, Akshay, Badawy, Sherif M., Lazarus, Hillard M., Hashem, Hasan, Szwajcer, David, Knight, Jennifer M., Bhatt, Neel S., Page, Kristin, Beattie, Sara, Arai, Yasuyuki, Liu, Hongtao, Arnold, Staci D., Freytes, César O., Abid, Muhammad Bilal, Beitinjaneh, Amer, Farhadfar, Nosha, Wirk, Baldeep, Winestone, Lena E., Agrawal, Vaibhav, Preussler, Jaime M., Seo, Sachiko, Hashmi, Shahrukh, Lehmann, Leslie, Wood, William A., Rangarajan, Hemalatha G., Saber, Wael, and Majhail, Navneet S.

Published
2023
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9. Neighborhood poverty and pediatric allogeneic hematopoietic cell transplantation outcomes: a CIBMTR analysis

Author

Bona, Kira, Brazauskas, Ruta, He, Naya, Lehmann, Leslie, Abdel-Azim, Hisham, Ahmed, Ibrahim A, Al-Homsi, A Samer, Aljurf, Mahmoud, Arnold, Staci D., Badawy, Sherif M, Battiwalla, Minoo, Beattie, Sara, Bhatt, Neel S., Dalal, Jignesh, Dandoy, Christopher E., Diaz, Miguel Angel, Frangoul, Haydar A., Freytes, César O., Ganguly, Siddhartha, George, Biju, Gomez-Almaguer, David, Hahn, Theresa, Kamble, Rammurti T., Knight, Jennifer M., LeMaistre, C. Fred, Law, Jason, Lazarus, Hillard M., Majhail, Navneet S., Olsson, Richard F., Preussler, Jaime, Savani, Bipin N., Schears, Raquel, Seo, Sachiko, Sharma, Akshay, Srivastava, Alok, Steinberg, Amir, Szwajcer, David, Wirk, Baldeep, Yoshimi, Ayami, Khera, Nandita, Wood, William A., Hashmi, Shahrukh, Duncan, Christine N., and Saber, Wael

Published
2021
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10. Household income and health‐related quality of life in children receiving treatment for acute myeloid leukemia: Potential impact of selection bias in health equity research.

Author

Newman, Haley, Li, Yimei, Huang, Yuan‐Shung V., Elgarten, Caitlin W., Myers, Regina M., Ruiz, Jenny, Zheng, Daniel J., Leahy, Alison Barz, Aftandilian, Catherine, Arnold, Staci D., Bona, Kira, Gramatges, M. Monica, Heneghan, Mallorie B., Maloney, Kelly W., Modi, Arunkumar J., Mody, Rajen J., Morgan, Elaine, Rubnitz, Jeffrey, Winick, Naomi, and Wilkes, Jennifer J.

Subjects
INCOME, QUALITY of life, ACUTE myeloid leukemia, HEALTH equity, CHILD patients
Abstract

Objective: Examine the influence of household income on health‐related quality of life (HRQOL) among children with newly diagnosed acute myeloid leukemia (AML). Design: Secondary analysis of data prospectively collected from pediatric patients receiving treatment for AML at 14 hospitals across the United States. Exposure: Household income was self‐reported on a demographic survey. The examined mediators included the acuity of presentation and treatment toxicity. Outcome: Caregiver proxy reported assessment of patient HRQOL from the Peds QL 4.0 survey. Result: Children with AML (n = 131) and caregivers were prospectively enrolled to complete PedsQL assessments. HRQOL scores were better for patients in the lowest versus highest income category (mean ± SD: 76.0 ± 14 household income <$25,000 vs. 59.9 ± 17 income ≥$75,000; adjusted mean difference: 11.2, 95% CI: 2.2–20.2). Seven percent of enrolled patients presented with high acuity (ICU‐level care in the first 72 h), and 16% had high toxicity (any ICU‐level care); there were no identifiable differences by income, refuting mediating roles in the association between income and HRQOL. Enrolled patients were less likely to be Black/African American (9.9% vs. 22.2%), more likely to be privately insured (50.4% vs. 40.7%), and more likely to have been treated on a clinical trial (26.7% vs. 18.5%) compared to eligible unenrolled patients not enrolled. Evaluations of potential selection bias on the association between income and HRQOL suggested differences in HRQOL may be smaller than observed or even in the opposing direction. Conclusions: While primary analyses suggested lower household income was associated with superior HRQOL, differential participation may have biased these results. Future studies should partner with patients/families to identify strategies for equitable participation in clinical research. [ABSTRACT FROM AUTHOR]

Published
2024
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11. The Impact of Donor Type on Outcomes and Cost of Allogeneic Hematopoietic Cell Transplantation for Pediatric Leukemia: A Merged Center for International Blood and Marrow Transplant Research and Pediatric Health Information System Analysis

Author

Arnold, Staci D., Brazauskas, Ruta, He, Naya, Li, Yimei, Hall, Matt, Atsuta, Yoshiko, Dalal, Jignesh, Hahn, Theresa, Khera, Nandita, Bonfim, Carmem, Hashmi, Shahrukh, Parsons, Susan, Wood, William A., Steinberg, Amir, Freytes, César O., Dandoy, Christopher E., Marks, David I., Lazarus, Hillard M., Abdel-Azim, Hisham, Bitan, Menachem, Diaz, Miguel Angel, Olsson, Richard F., Gergis, Usama, Seber, Adriana, Wirk, Baldeep, LeMaistre, C. Fred, Ustun, Celalettin, Duncan, Christine, Rizzieri, David, Szwajcer, David, Fagioli, Franca, Frangoul, Haydar, Knight, Jennifer M., Kamble, Rammurti T., Mehta, Paulette, Schears, Raquel, Satwani, Prakash, Pulsipher, Michael A., Aplenc, Richard, and Saber, Wael

Published
2020
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12. Comprehensive Prognostication in Critically Ill Pediatric Hematopoietic Cell Transplant Patients: Results from Merging the Center for International Blood and Marrow Transplant Research (CIBMTR) and Virtual Pediatric Systems (VPS) Registries

Author

Zinter, Matt S., Logan, Brent R., Fretham, Caitrin, Sapru, Anil, Abraham, Allistair, Aljurf, Mahmoud D., Arnold, Staci D., Artz, Andrew, Auletta, Jeffery J., Chhabra, Saurabh, Copelan, Edward, Duncan, Christine, Gale, Robert P., Guinan, Eva, Hematti, Peiman, Keating, Amy K., Marks, David I., Olsson, Richard, Savani, Bipin N., Ustun, Celalettin, Williams, Kirsten M., Pasquini, Marcelo C., and Dvorak, Christopher C.

Published
2020
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13. Epidemiology and Severity of Illness of MIS-C and Kawasaki Disease During the COVID-19 Pandemic

Author

Molloy, Matthew J., primary, Auger, Katherine A., additional, Hall, Matt, additional, Shah, Samir S., additional, Schondelmeyer, Amanda C., additional, Parikh, Kavita, additional, Kazmier, Katherine M., additional, Katragadda, Harita, additional, Jacob, Seethal A., additional, Jerardi, Karen E., additional, Ivancie, Rebecca, additional, Hartley, David, additional, Bryan, Mersine A., additional, Bhumbra, Samina, additional, Arnold, Staci D., additional, and Brady, Patrick W., additional

Published
2023
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14. Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance

Author

Stenger, Elizabeth, Xiang, Yijin, Wetzel, Martha, Gillespie, Scott, Chellapandian, Deepak, Shah, Rikin, Arnold, Staci D., Bhatia, Monica, Chaudhury, Sonali, Eckrich, Michael J., Kanter, Julie, Kasow, Kimberly A., Krajewski, Jennifer, Nickel, Robert S., Ngwube, Alexander I., Olson, Tim S., Rangarajan, Hemalatha G., Wobma, Holly, Guilcher, Gregory M.T., Horan, John T., Krishnamurti, Lakshmanan, Shenoy, Shalini, and Abraham, Allistair

Published
2023
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15. Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States: A Cost-Effectiveness Analysis.

Author

Basu, Anirban, Winn, Aaron N., Johnson, Kate M., Jiao, Boshen, Devine, Beth, Hankins, Jane S., Arnold, Staci D., Bender, M.A., and Ramsey, Scott D.

Subjects
SICKLE cell anemia, GENE therapy, BONE marrow cells, FETAL hemoglobin, ERYTHROCYTES, MEDICAID, DISEASE complications, PRIAPISM
Abstract

The U.S. Food and Drug Administration recently approved 2 genetic therapies for sickle cell disease. Both therapies increase the production of nonsickling red blood cells, either fetal hemoglobin or a gene therapy–derived hemoglobin similar to hemoglobin A. In addition, both therapies require collection of a patient's stem cells, modification of the cells in the laboratory, high-dose chemotherapy to remove existing bone marrow cells, and a single-dose infusion of the patient's modified stem cells. This article uses 2 different cost-effectiveness models to estimate whether these therapies provide enough value to justify their high costs. Visual Abstract. Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States: The U.S. Food and Drug Administration recently approved 2 genetic therapies for sickle cell disease. Both therapies increase the production of nonsickling red blood cells, either fetal hemoglobin or a gene therapy–derived hemoglobin similar to hemoglobin A. In addition, both therapies require collection of a patient's stem cells, modification of the cells in the laboratory, high-dose chemotherapy to remove existing bone marrow cells, and a single-dose infusion of the patient's modified stem cells. This article uses 2 different cost-effectiveness models to estimate whether these therapies provide enough value to justify their high costs. Background: Sickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage community. Objective: To evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs). Design: Comparative modeling analysis across 2 independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) using the same databases. Data Sources: Centers for Medicare & Medicaid Services claims data, 2008 to 2016; published literature. Target Population: Persons eligible for gene therapy. Time Horizon: Lifetime. Perspective: U.S. health care sector and societal. Intervention: Gene therapy versus common care. Outcome Measures: Incremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves. Results of Base-Case Analysis: At an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193 000 per QALY and $427 000 per QALY, respectively, under the health care sector perspective. Corresponding estimates from the societal perspective were $126 000 per QALY and $281 000 per QALY. The difference in results between models stemmed primarily from considering a slightly different target population and incorporating the quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome in the UW model. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on the use of alternative effective metrics or equity-informed threshold values. Results of Sensitivity Analysis: Results were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival. Limitation: The short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study. Conclusion: Gene therapy for SCD below a $2 million price tag is likely to be cost-effective when applying a societal perspective at an equity-informed threshold for cost-effectiveness analysis. Primary Funding Source: National Heart, Lung, and Blood Institute. [ABSTRACT FROM AUTHOR]

Published
2024
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17. Child and family perceptions of satisfaction with neutropenia management in pediatric acute myeloid leukemia

Author

Szymczak, Julia E., primary, Getz, Kelly D., additional, Madding, Rachel, additional, Shuster, Sydney, additional, Aftandilian, Catherine, additional, Arnold, Staci D., additional, Collier, Anderson B., additional, Gramatges, Maria M., additional, Henry, Meret, additional, Hijiya, Nobuko, additional, Mian, Amir, additional, Raetz, Elizabeth, additional, Fisher, Brian T., additional, and Aplenc, Richard, additional

Published
2023
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19. Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation

Author

Shenoy, Shalini, Angelucci, Emanuele, Arnold, Staci D., Baker, K. Scott, Bhatia, Monica, Bresters, Dorine, Dietz, Andrew C., De La Fuente, Josu, Duncan, Christine, Gaziev, Javid, King, Allison A., Pulsipher, Michael A., Smith, Angela R., and Walters, Mark C.

Published
2017
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23. Preliminary Results of Promis Sexual Function and Satisfaction and Perceived Risk for Infertility after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Stellar Study

Author

Meacham, Lillian R, primary, Veludhandi, Anirudh, additional, Xiang, Yijin, additional, Stenger, Elizabeth, additional, Arnold, Staci D., additional, Mehta, Akanksha, additional, Pecker, Lydia H., additional, Hsieh, Michael, additional, Maher, Jacqueline Y, additional, and Krishnamurti, Lakshmanan, additional

Published
2022
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25. Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study

Author

George, Sobenna A, primary, Veludhandi, Anirudh, additional, Xiang, Yijin, additional, Stenger, Elizabeth, additional, Arnold, Staci D., additional, Mehta, Akanksha, additional, Schirmer, David A, additional, Spencer, Jessica B, additional, Guilcher, Gregory M, additional, Bhatia, Monica, additional, Abraham, Allistair, additional, Gomez-Lobo, Veronica, additional, Krishnamurti, Lakshmanan, additional, and Meacham, Lillian R, additional

Published
2022
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26. Influence of Household Income on Health-Related Quality of Life in Children with Acute Myeloid Leukemia

Author

Newman, Haley, primary, Li, Yimei, additional, Huang, Yuan-Shung V., additional, Elgarten, Caitlin W, additional, Myers, Regina M., additional, Ruiz, Jenny, additional, Leahy, Allison Barz, additional, Aftandilian, Catherine, additional, Arnold, Staci D., additional, Bona, Kira O, additional, Heneghan, Mallorie M., additional, Gramatges, M Monica, additional, Maloney, Kelly W, additional, Modi, Arunkumar J., additional, Mody, Rajen, additional, Winick, Naomi J, additional, Morgan, Elaine, additional, Wilkes, Jennifer J., additional, Seif, Alix E., additional, Fisher, Brian T., additional, Aplenc, Richard, additional, and Getz, Kelly D., additional

Published
2022
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27. A New Phen X Collection: Sickle Cell Disease Psychosocial and Social Determinants of Health Protocols

Author

Crosby, Lori E., primary, King, Allison A., additional, Porter, Jerlym S., additional, Badawy, Sherif M., additional, Bulgin, Dominique, additional, Hardy, Steven J., additional, Arnold, Staci D., additional, Williford, Desiree, additional, Treadwell, Marsha, additional, Huggins, Wayne, additional, Ives, Cataia, additional, Sullivan, Marian, additional, and Hamilton, Carol, additional

Published
2022
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29. Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study

Author

Krishnamurti, Lakshmanan, primary, Arnold, Staci D, additional, Haight, Ann, additional, Abraham, Allistair, additional, Guilcher, Gregory MT, additional, John, Tami, additional, Bakshi, Nitya, additional, Shenoy, Shalini, additional, Syrjala, Karen, additional, Martin, Paul L, additional, Chaudhury, Sonali, additional, Eames, Gretchen, additional, Olowoselu, Olusola Festus, additional, Hsieh, Matthew, additional, De La Fuente, Josu, additional, Kasow, Kimberly A, additional, Stenger, Elizabeth, additional, Mertens, Anne, additional, El-Rassi, Fuad, additional, Lane, Peter, additional, Shaw, Bronwen E, additional, Meacham, Lillian, additional, and Archer, David, additional

Published
2022
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31. Risk of bacterial bloodstream infection does not vary by central-line type during neutropenic periods in pediatric acute myeloid leukemia

Author

Elgarten, Caitlin W., primary, Otto, William R., additional, Shenton, Luke, additional, Stein, Madison T., additional, Horowitz, Joseph, additional, Aftandilian, Catherine, additional, Arnold, Staci D., additional, Bona, Kira O., additional, Caywood, Emi, additional, Collier, Anderson B., additional, Gramatges, M. Monica, additional, Henry, Meret, additional, Lotterman, Craig, additional, Maloney, Kelly, additional, Modi, Arunkumar J., additional, Mian, Amir, additional, Mody, Rajen, additional, Morgan, Elaine, additional, Raetz, Elizabeth A., additional, Verma, Anupam, additional, Winick, Naomi, additional, Wilkes, Jennifer J., additional, Yu, Jennifer C., additional, Aplenc, Richard, additional, Fisher, Brian T., additional, and Getz, Kelly D., additional

Published
2022
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32. Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study (Preprint)

Author

Krishnamurti, Lakshmanan, primary, Arnold, Staci D, additional, Haight, Ann, additional, Abraham, Allistair, additional, Guilcher, Gregory MT, additional, John, Tami, additional, Bakshi, Nitya, additional, Shenoy, Shalini, additional, Syrjala, Karen, additional, Martin, Paul L, additional, Chaudhury, Sonali, additional, Eames, Gretchen, additional, Olowoselu, Olusola Festus, additional, Hsieh, Matthew, additional, De La Fuente, Josu, additional, Kasow, Kimberly A, additional, Stenger, Elizabeth, additional, Mertens, Anne, additional, El-Rassi, Fuad, additional, Lane, Peter, additional, Shaw, Bronwen E, additional, Meacham, Lillian, additional, and Archer, David, additional

Published
2022
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33. Contributors

Author

Al-Ghafry, Maha, Alme, Charlotte, Alobeid, Bachir, Arnold, Staci D., Askew, Megan, Beauchemin, Melissa P., Bhatia, Monica, Bombacie, Michelle, Bonilla, Mary Ann, Brittenham, Gary M., Broglie, Larisa, Chen, Jing, Connolly, Eileen, Constantino, Caitlin, Davis, Laurie, Debelenko, Larisa, Dela Cruz, Filemon S., El-Mallawany, Nader Kim, Ender, Katie, Fallon, Erica M., Foca, Marc D., Gallitto, Matthew, Gampel, Bradley, Gartrell, Robyn D., Garvin, James, Jr, Gass, David, George, Gabriella E., Glade Bender, Julia, Glasser, Chana L., Graham, Rudi-Ann, Granowetter, Linda, Green, Nancy S., Gulati, Nitya, Hijiya, Nobuko, Hochberg, Jess, Joffe, Lenat, Kadenhe-Chiweshe, Angela V., Kahn, Justine M., Kaicker, Shipra, Kaur, Dominder, Kelly, Kara M., Kinnaman, Michael D., Kochhar, Manpreet, Krajewski, Jennifer, Kurtz, Justin L., Ladas, Elena J., Levine, Jennifer, Lee, Margaret T., Lieb, Kristin, Madubata, Chinwe, Mansukhani, Mahesh M., Marks, Lianna J., McGuinn, Catherine, Menell, Jill S., Myers, Regina, Minns, Hanna E., Moisander Joyce, Hanna, Neunert, Cindy, Oberg, Jennifer, Orjuela, Manuela, Parrott, Andrew Myles, Pavisic, Jovana, Phillips, Lia N., Ramirez, Evelyn M., Ricci, Angela, Rosenzweig, Jaclyn D., Satwani, Prakash, Silverman, Andrew M., Steinberg, Dara M., Sheth, Sujit, Sulis, Maria Luisa, Szalontay, Luca, Tang, Amy, Tomb, Meghan F., Varkey, Joyce, Webster Carrion, Andrea, Weiner, Michael, Weisberg, Stuart P., Wu, Cheng-Chia, Yamashiro, Darrell J., and Zacharoulis, Stergios

Published
2024
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34. Long-Term Quality of Life after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from the Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry (STELLAR)

Author

Arnold, Staci D., primary, Bakshi, Nitya, additional, Chang, Zhao, additional, Ross, Diana, additional, Abraham, Allistair, additional, Bhatia, Monica, additional, Guilcher, Gregory M.T., additional, Haight, Ann, additional, Hsieh, Matthew, additional, Kasow, Kimberly A., additional, Lane, Peter A., additional, Meacham, Lillian, additional, Olowoselu, Festus Olusola, additional, and Krishnamurti, Lakshmanan, additional

Published
2021
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35. Mobility and Muscle Strength in Recipients of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from Sickle Transplant Evaluation of Longterm and Late Effects Registry (STELLaR)

Author

Veludhandi, Anirudh, primary, John, Tami D, additional, Bakshi, Nitya, additional, Arnold, Staci D., additional, Bhatia, Monica, additional, Lane, Peter A., additional, Meacham, Lillian, additional, Khemani, Kirshma, additional, Chaudhury, Sonali, additional, Olowoselu, Festus Olusola, additional, Kasow, Kimberly A., additional, Hsieh, Matthew, additional, Abraham, Allistair, additional, Guilcher, Gregory M.T., additional, and Krishnamurti, Lakshmanan, additional

Published
2021
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36. Medical Outcomes, Quality of Life, and Family Perceptions for Outpatient vs Inpatient Neutropenia Management After Chemotherapy for Pediatric Acute Myeloid Leukemia

Author

Getz, Kelly D., primary, Szymczak, Julia E., additional, Li, Yimei, additional, Madding, Rachel, additional, Huang, Yuan-Shung V., additional, Aftandilian, Catherine, additional, Arnold, Staci D., additional, Bona, Kira O., additional, Caywood, Emi, additional, Collier, Anderson B., additional, Gramatges, M. Monica, additional, Henry, Meret, additional, Lotterman, Craig, additional, Maloney, Kelly, additional, Mian, Amir, additional, Mody, Rajen, additional, Morgan, Elaine, additional, Raetz, Elizabeth A., additional, Rubnitz, Jeffrey, additional, Verma, Anupam, additional, Winick, Naomi, additional, Wilkes, Jennifer J., additional, Yu, Jennifer C., additional, Fisher, Brian T., additional, and Aplenc, Richard, additional

Published
2021
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37. Risk of bacterial bloodstream infection does not vary by central-line type during neutropenic periods in pediatric acute myeloid leukemia

Author

Elgarten, Caitlin W., Otto, William R., Shenton, Luke, Stein, Madison T., Horowitz, Joseph, Aftandilian, Catherine, Arnold, Staci D., Bona, Kira O., Caywood, Emi, Collier, Anderson B., Gramatges, M. Monica, Henry, Meret, Lotterman, Craig, Maloney, Kelly, Modi, Arunkumar J., Mian, Amir, Mody, Rajen, Morgan, Elaine, Raetz, Elizabeth A., Verma, Anupam, Winick, Naomi, Wilkes, Jennifer J., Yu, Jennifer C., Aplenc, Richard, Fisher, Brian T., and Getz, Kelly D.

Abstract

AbstractBackground:Bloodstream infections (BSIs) are a frequent cause of morbidity in patients with acute myeloid leukemia (AML), due in part to the presence of central venous access devices (CVADs) required to deliver therapy.Objective:To determine the differential risk of bacterial BSI during neutropenia by CVAD type in pediatric patients with AML.Methods:We performed a secondary analysis in a cohort of 560 pediatric patients (1,828 chemotherapy courses) receiving frontline AML chemotherapy at 17 US centers. The exposure was CVAD type at course start: tunneled externalized catheter (TEC), peripherally inserted central catheter (PICC), or totally implanted catheter (TIC). The primary outcome was course-specific incident bacterial BSI; secondary outcomes included mucosal barrier injury (MBI)-BSI and non-MBI BSI. Poisson regression was used to compute adjusted rate ratios comparing BSI occurrence during neutropenia by line type, controlling for demographic, clinical, and hospital-level characteristics.Results:The rate of BSI did not differ by CVAD type: 11 BSIs per 1,000 neutropenic days for TECs, 13.7 for PICCs, and 10.7 for TICs. After adjustment, there was no statistically significant association between CVAD type and BSI: PICC incident rate ratio [IRR] = 1.00 (95% confidence interval [CI], 0.75–1.32) and TIC IRR = 0.83 (95% CI, 0.49–1.41) compared to TEC. When MBI and non-MBI were examined separately, results were similar.Conclusions:In this large, multicenter cohort of pediatric AML patients, we found no difference in the rate of BSI during neutropenia by CVAD type. This may be due to a risk-profile for BSI that is unique to AML patients.

Published
2023
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38. Medical Outcomes, Quality of Life, and Family Perceptions for Outpatient vs Inpatient Neutropenia Management After Chemotherapy for Pediatric Acute Myeloid Leukemia

Author

Getz, Kelly D, Szymczak, Julia E, Li, Yimei, Madding, Rachel, Huang, Yuan-Shung V, Aftandilian, Catherine, Arnold, Staci D, Bona, Kira O, Caywood, Emi, Collier, Anderson B, Gramatges, M Monica, Henry, Meret, Lotterman, Craig, Maloney, Kelly, Mian, Amir, Mody, Rajen, Morgan, Elaine, Raetz, Elizabeth A, Rubnitz, Jeffrey, Verma, Anupam, Winick, Naomi, Wilkes, Jennifer J, Yu, Jennifer C, Fisher, Brian T, Aplenc, Richard, Getz, Kelly D, Szymczak, Julia E, Li, Yimei, Madding, Rachel, Huang, Yuan-Shung V, Aftandilian, Catherine, Arnold, Staci D, Bona, Kira O, Caywood, Emi, Collier, Anderson B, Gramatges, M Monica, Henry, Meret, Lotterman, Craig, Maloney, Kelly, Mian, Amir, Mody, Rajen, Morgan, Elaine, Raetz, Elizabeth A, Rubnitz, Jeffrey, Verma, Anupam, Winick, Naomi, Wilkes, Jennifer J, Yu, Jennifer C, Fisher, Brian T, and Aplenc, Richard

Abstract

Importance: Pediatric acute myeloid leukemia (AML) requires multiple courses of intensive chemotherapy that result in neutropenia, with significant risk for infectious complications. Supportive care guidelines recommend hospitalization until neutrophil recovery. However, there are little data to support inpatient over outpatient management. Objective: To evaluate outpatient vs inpatient neutropenia management for pediatric AML. Design, setting, and participants: This cohort study used qualitative and quantitative methods to compare medical outcomes, patient health-related quality of life (HRQOL), and patient and family perceptions between outpatient and inpatient neutropenia management. The study included patients from 17 US pediatric hospitals with frontline chemotherapy start dates ranging from January 2011 to July 2019, although the specific date ranges differed for the individual analyses by design and relative timing. Data were analyzed from August 2019 to February 2020. Exposures: Discharge to outpatient vs inpatient neutropenia management. Main outcomes and measures: The primary outcomes of interest were course-specific bacteremia incidence, times to next course, and patient HRQOL. Course-specific mortality was a secondary medical outcome. Results: Primary quantitative analyses included 554 patients (272 [49.1%] girls and 282 [50.9%] boys; mean [SD] age, 8.2 [6.1] years). Bacteremia incidence was not significantly different during outpatient vs inpatient management (67 courses [23.8%] vs 265 courses [29.0%]; adjusted rate ratio, 0.73; 95% CI, 0.56 to 1.06; P = .08). Outpatient management was not associated with delays to the next course compared with inpatient management (mean [SD] 30.7 [12.2] days vs 32.8 [9.7] days; adjusted mean difference, -2.2; 95% CI, -4.1 to -0.2, P = .03). Mortality during intensification II was higher for patients who received outpatient management compared with those who received inpatient management (3 patients [5.4%] vs 1 patient

Published
2021

42. Home or Away from Home: A Multi-Institution Study Comparing Medical Outcomes, Patient Perspectives, and Health-Related Quality of Life for Outpatient Versus Inpatient Management after Chemotherapy for Pediatric Acute Myeloid Leukemia

Author

Getz, Kelly D, primary, Li, Yimei, primary, Szymczak, Julia E, primary, Aftandilian, Catherine, primary, Arnold, Staci D., primary, Bona, Kira O, primary, Caywood, Emi, primary, Collier, Anderson B., primary, Gramatges, Maria Monica Monica, primary, Henry, Meret, primary, Lotterman, Craig, primary, Maloney, Kelly, primary, Mian, Amir, primary, Mody, Rajen, primary, Raetz, Elizabeth A., primary, Rubnitz, Jeffrey, primary, Verma, Anupam, primary, Winick, Naomi, primary, Wilkes, Jennifer J., primary, Yu, Jennifer C., primary, Fisher, Brian T., primary, and Aplenc, Richard, primary

Published
2019
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43. 108 - Improved Mortality Prognostication for Critically Ill Pediatric Hematopoietic Cell Transplant Patients: Results From a Virtual Pediatric Systems (VPS) and Center for International Blood and Marrow Transplant Research (CIBMTR) Database Merger

Author

Zinter, Matt S., Logan, Brent R., Zhu, Xiaochun, Sapru, Anil, Abraham, Allistair, Aljurf, Mahmoud D., Arnold, Staci D., Artz, Andrew, Auletta, Jeffery J., Chhabra, Saurabh, Copelan, Edward, Duncan, Christine, Fretham, Caitrin, Gale, Robert Peter, Guinan, Eva, Hematti, Peiman, Keating, Amy K., Marks, David I., Savani, Bipin N., Olsson, Richard, Ustun, Celalettin, Williams, Kirsten M., Pasquini, Marcelo C., and Dvorak, Christopher C.

Published
2018
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45. Improved Mortality Prognostication for Critically Ill Pediatric Hematopoietic Cell Transplant Patients: Results From a Virtual Pediatric Systems (VPS) and Center for International Blood and Marrow Transplant Research (CIBMTR) Database Merger

Author

Zinter, Matt S., primary, Logan, Brent R., additional, Zhu, Xiaochun, additional, Sapru, Anil, additional, Abraham, Allistair, additional, Aljurf, Mahmoud D., additional, Arnold, Staci D., additional, Artz, Andrew, additional, Auletta, Jeffery J., additional, Chhabra, Saurabh, additional, Copelan, Edward, additional, Duncan, Christine, additional, Fretham, Caitrin, additional, Gale, Robert Peter, additional, Guinan, Eva, additional, Hematti, Peiman, additional, Keating, Amy K., additional, Marks, David I., additional, Savani, Bipin N., additional, Olsson, Richard, additional, Ustun, Celalettin, additional, Williams, Kirsten M., additional, Pasquini, Marcelo C., additional, and Dvorak, Christopher C., additional

Published
2018
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46. Identifying patient- and family-centered outcomes relevant to inpatient versus at-home management of neutropenia in children with acute myeloid leukemia

Author

Szymczak, Julia E., primary, Getz, Kelly D., additional, Madding, Rachel, additional, Fisher, Brian, additional, Raetz, Elizabeth, additional, Hijiya, Nobuko, additional, Gramatges, Maria M., additional, Henry, Meret, additional, Mian, Amir, additional, Arnold, Staci D., additional, Aftandilian, Catherine, additional, Collier, Anderson B., additional, and Aplenc, Richard, additional

Published
2017
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47. Association of Guideline-Adherent Antibiotic Treatment With Readmission of Children With Sickle Cell Disease Hospitalized With Acute Chest Syndrome

Author

Bundy, David G., primary, Richardson, Troy E., additional, Hall, Matthew, additional, Raphael, Jean L., additional, Brousseau, David C., additional, Arnold, Staci D., additional, Kalpatthi, Ram V., additional, Ellison, Angela M., additional, Oyeku, Suzette O., additional, and Shah, Samir S., additional

Published
2017
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48. Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

Author

Arnold, Staci D., Brazauskas, Ruta, He, Naya, Li, Yimei, Aplenc, Richard, Jin, Zhezhen, Hall, Matt, Atsuta, Yoshiko, Dalal, Jignesh, Hahn, Theresa, Khera, Nandita, Bonfim, Carmem, Majhail, Navneet S., Diaz, Miguel Angel, Freytes, Cesar O., Wood, William A., Savani, Bipin N., Kamble, Rammurti T., Parsons, Susan, Ahmed, Ibrahim, Sullivan, Keith, Beattie, Sara, Dandoy, Christopher, Munker, Reinhold, Marino, Susana, Bitan, Menachem, Abdel-Azim, Hisham, Aljurf, Mahmoud, Olsson, Richard F., Joshi, Sarita, Buchbinder, Dave, Eckrich, Michael J., Hashmi, Shahrukh, Lazarus, Hillard, Marks, David I., Steinberg, Amir, Saad, Ayman, Gergis, Usama, Krishnamurti, Lakshmanan, Abraham, Allistair, Rangarajan, Hemalatha G., Walters, Mark, Lipscomb, Joseph, Saber, Wael, Satwani, Prakash, Arnold, Staci D., Brazauskas, Ruta, He, Naya, Li, Yimei, Aplenc, Richard, Jin, Zhezhen, Hall, Matt, Atsuta, Yoshiko, Dalal, Jignesh, Hahn, Theresa, Khera, Nandita, Bonfim, Carmem, Majhail, Navneet S., Diaz, Miguel Angel, Freytes, Cesar O., Wood, William A., Savani, Bipin N., Kamble, Rammurti T., Parsons, Susan, Ahmed, Ibrahim, Sullivan, Keith, Beattie, Sara, Dandoy, Christopher, Munker, Reinhold, Marino, Susana, Bitan, Menachem, Abdel-Azim, Hisham, Aljurf, Mahmoud, Olsson, Richard F., Joshi, Sarita, Buchbinder, Dave, Eckrich, Michael J., Hashmi, Shahrukh, Lazarus, Hillard, Marks, David I., Steinberg, Amir, Saad, Ayman, Gergis, Usama, Krishnamurti, Lakshmanan, Abraham, Allistair, Rangarajan, Hemalatha G., Walters, Mark, Lipscomb, Joseph, Saber, Wael, and Satwani, Prakash

Abstract

Advances in allogeneic hematopoietic cell transplantation for sickle cell disease have improved outcomes, but there is limited analysis of healthcare utilization in this setting. We hypothesized that, compared to late transplantation, early transplantation (at age < 10 years) improves outcomes and decreases healthcare utilization. We performed a retrospective study of children transplanted for sickle cell disease in the USA during 2000-2013 using two large databases. Univariate and Cox models were used to estimate associations of demographics, sickle cell disease severity, and transplant-related variables with mortality and chronic graft-versus-host disease, while Wilcoxon, Kruskal-Wallis, or linear trend tests were applied for the estimates of healthcare utilization. Among 161 patients with a 2-year overall survival rate of 90% (95% confidence interval [CI] 85-95%) mortality was significantly higher in those who underwent late transplantation versus early (hazard ratio (HR) 21, 95% CI 2.8-160.8, P=0.003) and unrelated compared to matched sibling donor transplantation (HR 5.9, 95% CI 1.7-20.2, P=0.005). Chronic graftversus host disease was significantly more frequent among those translanted late (HR 1.9, 95% CI 1.0-3.5, P=0.034) and those who received an unrelated graft (HR 2.5, 95% CI 1.2-5.4; P=0.017). Merged data for 176 patients showed that the median total adjusted transplant cost per patient was $467,747 (range: $344,029-$ 799,219). Healthcare utilization was lower among recipients of matched sibling donor grafts and those with low severity disease compared to those with other types of donor and disease severity types (P<0.001 and P=0.022, respectively); no association was demonstrated with late transplantation (P=0.775). Among patients with 2-year pre-and post-transplant data (n=41), early transplantation was associated with significant reductions in admissions (P<0.001), length of stay (P<0.001), and cost (P=0.008). Early transplant outcomes need

Published
2017
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49. Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

Author

Arnold, Staci D., primary, Brazauskas, Ruta, additional, He, Naya, additional, Li, Yimei, additional, Aplenc, Richard, additional, Jin, Zhezhen, additional, Hall, Matt, additional, Atsuta, Yoshiko, additional, Dalal, Jignesh, additional, Hahn, Theresa, additional, Khera, Nandita, additional, Bonfim, Carmem, additional, Majhail, Navneet S., additional, Diaz, Miguel Angel, additional, Freytes, Cesar O., additional, Wood, William A., additional, Savani, Bipin N., additional, Kamble, Rammurti T., additional, Parsons, Susan, additional, Ahmed, Ibrahim, additional, Sullivan, Keith, additional, Beattie, Sara, additional, Dandoy, Christopher, additional, Munker, Reinhold, additional, Marino, Susana, additional, Bitan, Menachem, additional, Abdel-Azim, Hisham, additional, Aljurf, Mahmoud, additional, Olsson, Richard F., additional, Joshi, Sarita, additional, Buchbinder, Dave, additional, Eckrich, Michael J., additional, Hashmi, Shahrukh, additional, Lazarus, Hillard, additional, Marks, David I., additional, Steinberg, Amir, additional, Saad, Ayman, additional, Gergis, Usama, additional, Krishnamurti, Lakshmanan, additional, Abraham, Allistair, additional, Rangarajan, Hemalatha G., additional, Walters, Mark, additional, Lipscomb, Joseph, additional, Saber, Wael, additional, and Satwani, Prakash, additional

Published
2017
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50. Role of Donor Source on Clinical Outcomes and Inpatient Resource Utilization for Hematopoietic Cell Transplantation in Children with Acute Leukemia

Author

Arnold, Staci D, primary, Brazauskas, Ruta, additional, He, Naya, additional, Li, Yimei, additional, Satwani, Prakash, additional, Pulsipher, Michael A, additional, Hall, Matt, additional, Atsuta, Yoshiko, additional, Dalal, Jignesh D, additional, Hahn, Theresa, additional, Bonfim, Carmem, additional, Khera, Nandita, additional, Aplenc, Richard, additional, and Saber, Wael, additional

Published
2016
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