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Article ; Online: Plastic bronchitis: Autopsy findings in the sudden death of a healthy pediatric patient with sickle cell disease.

Saunders, Katherine / Garrett, Erin / Crosby, Ashley / Park, Yara / Kasow, Kimberly / Moylan, Vincent / Thorne, Leigh

2022 Volume 69, Issue 8, Page(s) e29584

MeSH term(s)	Anemia, Sickle Cell/complications ; Autopsy ; Bronchitis ; Cause of Death ; Child ; Death, Sudden/etiology ; Humans ; Plastics
Chemical Substances	Plastics
Language	English
Publishing date	2022-02-07
Publishing country	United States
Document type	Letter
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.29584
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Article ; Online: Current and Future Cell Therapy Standards and Guidelines.

Atkins, J Wade / West, Kamille / Kasow, Kimberly A

Hematology/oncology clinics of North America

2019 Volume 33, Issue 5, Page(s) 839–855

Abstract: Cell biology researchers, cellular engineers, and clinicians are teaming together to create powerful drugs. The use of cell-derived products as biologics is rapidly advancing. These human cell-based products have great potential for treating serious ... ...

Abstract	Cell biology researchers, cellular engineers, and clinicians are teaming together to create powerful drugs. The use of cell-derived products as biologics is rapidly advancing. These human cell-based products have great potential for treating serious conditions but may have unidentified risks. Manipulations, expansions, and gene modifications increase the risks of unexpected outcomes. Implementation of the 21st Century Cures Act is opening avenues for accelerated approvals of these drugs for use in clinical trials and licensure. Although overwhelming, collaboration between regulators, industry, and research and medical communities enables the field to safely meet the needs of critically ill patients.
MeSH term(s)	Biological Products ; Cell- and Tissue-Based Therapy/methods ; Cell- and Tissue-Based Therapy/standards ; Cell- and Tissue-Based Therapy/trends ; Government Regulation ; Health Policy ; Humans ; Practice Guidelines as Topic/standards ; United States
Chemical Substances	Biological Products
Language	English
Publishing date	2019-07-30
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	93115-9
ISSN	1558-1977 ; 0889-8588
ISSN (online)	1558-1977
ISSN	0889-8588
DOI	10.1016/j.hoc.2019.05.008
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Article ; Online: Financial burden and recommended multilevel solutions among caregivers of pediatric hematopoietic stem cell transplant recipients.

Waters, Austin R / Biddell, Caitlin B / Killela, Mary / Kasow, Kimberly A / Page, Kristin / Wheeler, Stephanie B / Drier, Sarah W / Kelly, Matthew S / Robles, Joanna / Spees, Lisa P

Pediatric blood & cancer

2023 Volume 70, Issue 12, Page(s) e30700

Abstract: Background: The healthcare costs of patients who receive hematopoietic stem cell transplantation (HSCT) are substantial. At the same time, the increasing use of pediatric HSCT leaves more caregivers of pediatric HSCT recipients at risk for financial ... ...

Abstract	Background: The healthcare costs of patients who receive hematopoietic stem cell transplantation (HSCT) are substantial. At the same time, the increasing use of pediatric HSCT leaves more caregivers of pediatric HSCT recipients at risk for financial burden-an understudied area of research. Methods: Financial burden experienced by caregivers of recipients who received autologous or allogeneic transplants was assessed using an explanatory mixed-methods design including a one-time survey and semi-structured interviews. Financial burden was assessed through an adapted COmprehensive Score for financial Toxicity (COST) as well as questions about the types of out-of-pocket costs and cost-coping behaviors. Chi-squared or Fisher's exact tests were used to assess differences in costs incurred and coping behaviors by financial toxicity and financial toxicity by demographic factors. Interviews were audio recorded, transcribed, and analyzed using directed content analysis. Results: Of 99 survey participants, 64% experienced high financial toxicity (COST Conclusions: Considering the substantial, long-term financial burden among pediatric HSCT patients and their caregivers, this population would benefit from adapted and tailored financial burden interventions.
MeSH term(s)	Humans ; Child ; Financial Stress ; Caregivers ; Health Care Costs ; Health Expenditures ; Hematopoietic Stem Cell Transplantation
Language	English
Publishing date	2023-09-30
Publishing country	United States
Document type	Journal Article
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.30700
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Article ; Online: Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists' practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study.

Meier, Emily Riehm / Abraham, Allistair A / Ngwube, Alex / Janson, Isaac A / Guilcher, Gregory M T / Horan, John / Kasow, Kimberly A

Pediatric blood & cancer

2021 Volume 68, Issue 3, Page(s) e28861

Abstract: Background: Hematopoietic stem cell transplantation (HSCT) provides a curative therapy for children severely affected by sickle cell disease (SCD). Rejection-free survival after matched sibling donor (MSD) HSCT is very high, but adoption of HSCT as a ... ...

Abstract	Background: Hematopoietic stem cell transplantation (HSCT) provides a curative therapy for children severely affected by sickle cell disease (SCD). Rejection-free survival after matched sibling donor (MSD) HSCT is very high, but adoption of HSCT as a curative SCD therapy has been slow. In this study, we assess providers' perceptions about MSD HSCT for children with variable SCD severity, and determine the influence of provider characteristics on HSCT referrals. Procedure: After our Institutional Review Board deemed the study exempt, American Society of Pediatric Hematology/Oncology Clinical Forum listserv subscribers and American Society of Hematology members who self-identified as pediatric hematologists/oncologists (PHO) were emailed a survey. Analysis was performed to describe and evaluate correlations between participant demographics (including practice focus within PHO) and likelihood of HSCT referral for each scenario. Results: Spearman's rank correlation analysis did not reveal any significant relationship between demographic characteristics except practice focus and likelihood to refer to HSCT for any scenarios. Providers focused on SCD and HSCT were more likely to refer a child who had never been admitted to the hospital or had suboptimal adherence to hydroxyurea than general PHOs. A significantly higher proportion of all respondents would refer a child with β-thalassemia major (87%) than an asymptomatic child with HbSS (47%, P < .00001) or non-HbSS variant (23%, P < .00001). Conclusion: PSCD and HSCT physicians are more likely to refer for MSD HSCT in almost every condition than general PHO practitioners, likely because of increased awareness of long-term effects of SCD and safety of MSD HSCT for children with SCD.
MeSH term(s)	Adult ; Anemia, Sickle Cell/therapy ; Child ; Female ; Follow-Up Studies ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Male ; Middle Aged ; Oncologists/statistics & numerical data ; Practice Patterns, Physicians'/statistics & numerical data ; Prognosis ; Referral and Consultation/statistics & numerical data ; Surveys and Questionnaires
Language	English
Publishing date	2021-01-06
Publishing country	United States
Document type	Journal Article
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.28861
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Article ; Online: Anti-CD30 CAR T cells as consolidation after autologous haematopoietic stem-cell transplantation in patients with high-risk CD30

The Lancet. Haematology

2024

Abstract: Background: Chimeric antigen receptor (CAR) T cells targeting CD30 are safe and have promising activity when preceded by lymphodepleting chemotherapy. We aimed to determine the safety of anti-CD30 CAR T cells as consolidation after autologous ... ...

Abstract	Background: Chimeric antigen receptor (CAR) T cells targeting CD30 are safe and have promising activity when preceded by lymphodepleting chemotherapy. We aimed to determine the safety of anti-CD30 CAR T cells as consolidation after autologous haematopoietic stem-cell transplantation (HSCT) in patients with CD30 Methods: This phase 1 dose-escalation study was performed at two sites in the USA. Patients aged 3 years and older, with classical Hodgkin lymphoma or non-Hodgkin lymphoma with CD30 Findings: Between June 7, 2016, and Nov 30, 2020, 21 patients were enrolled and 18 patients (11 with Hodgkin lymphoma, six with T-cell lymphoma, one with grey zone lymphoma) were infused with anti-CD30 CAR T cells at a median of 22 days (range 16-44) after autologous HSCT. There were no dose-limiting toxicities observed, so the highest dose tested, 2 × 10 Interpretation: Anti-CD30 CAR T-cell infusion as consolidation after BEAM and autologous HSCT is safe, with low rates of toxicity and encouraging preliminary activity in patients with Hodgkin lymphoma at high risk of relapse, highlighting the need for larger studies to confirm these findings. Funding: National Heart Lung and Blood Institute, University Cancer Research Fund at the Lineberger Comprehensive Cancer Center.
Language	English
Publishing date	2024-03-28
Publishing country	England
Document type	Journal Article
ISSN	2352-3026
ISSN (online)	2352-3026
DOI	10.1016/S2352-3026(24)00064-4
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Article ; Online: Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.

American journal of hematology

2024 Volume 99, Issue 4, Page(s) 785–788

MeSH term(s)	Humans ; Unrelated Donors ; Bone Marrow Transplantation ; Hematopoietic Stem Cell Transplantation ; Anemia, Sickle Cell/therapy ; Graft vs Host Disease
Language	English
Publishing date	2024-02-11
Publishing country	United States
Document type	Letter
ZDB-ID	196767-8
ISSN	1096-8652 ; 0361-8609
ISSN (online)	1096-8652
ISSN	0361-8609
DOI	10.1002/ajh.27251
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Zs.A 1251: Show issues

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Article ; Online: Understanding the financial and psychological impact of employment disruption among caregivers of pediatric HSCT recipients: a mixed methods analysis.

Biddell, Caitlin B / Kasow, Kimberly A / Killela, Mary K / Page, Kristin M / Wheeler, Stephanie B / Drier, Sarah W / Kelly, Matthew S / Robles, Joanna M / Spees, Lisa P

Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer

2022 Volume 30, Issue 6, Page(s) 4747–4757

Abstract: Purpose: Pediatric hematopoietic stem cell transplantation (HSCT) confers a substantial financial burden onto patients' families. In addition to high direct medical costs, HSCTs typically require at least one caregiver to take time away from work or ... ...

Abstract	Purpose: Pediatric hematopoietic stem cell transplantation (HSCT) confers a substantial financial burden onto patients' families. In addition to high direct medical costs, HSCTs typically require at least one caregiver to take time away from work or other responsibilities, often leading to reduced household income. Using mixed methods, we sought to understand the impact of pediatric HSCT on caregiver employment and financial need. Methods: We surveyed caregivers of living pediatric patients who underwent HSCT at one of two southeastern transplant centers between 2012 and 2018 (N = 95). We then interviewed a subset of caregivers (N = 18) to understand whether and how employment disruption contributed to financial distress. Results: Among caregivers surveyed, the majority of household wage earners changed their work schedules to attend medical appointments and missed workdays. This resulted in income loss for 87% of families, with 31% experiencing an income reduction of over 50%. Qualitative interviews pointed to four emergent themes: (1) employment disruption exacerbated existing financial challenges; (2) parental division of labor between caregiving and providing financially led to heightened psychological distress; (3) existing employment leave and protection resources were essential but not sufficient; and (4) the ability to work remotely and having a supportive employer facilitated employment maintenance throughout the HSCT process. Conclusion: Expanded employment protections and access to accommodations are needed to limit the impact of HSCT on household income, health insurance, and financial hardship. Additionally, interventions are needed to ensure caregivers are equipped with the information necessary to navigate conversations with employers and prepare for the financial and psychological reality of employment disruption.
MeSH term(s)	Caregivers/psychology ; Child ; Employment ; Financial Stress ; Hematopoietic Stem Cell Transplantation ; Humans ; Income
Language	English
Publishing date	2022-02-07
Publishing country	Germany
Document type	Journal Article
ZDB-ID	1134446-5
ISSN	1433-7339 ; 0941-4355
ISSN (online)	1433-7339
ISSN	0941-4355
DOI	10.1007/s00520-022-06883-0
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Zs.A 3697: Show issues

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Article ; Online: Staphylococcus aureus Bloodstream Infection Due to Contaminated Hematopoietic Stem-Cell Graft.

Willis, Zachary I / Brondon, Jennifer E / Sickbert-Bennett, Emily E / Kasow, Kimberly A / Weber, David J

Infection control and hospital epidemiology

2018 Volume 39, Issue 3, Page(s) 367–369

MeSH term(s)	Adolescent ; Anti-Bacterial Agents/therapeutic use ; Bone Neoplasms/surgery ; Catheter-Related Infections/drug therapy ; Catheter-Related Infections/microbiology ; Cross Infection/drug therapy ; Cross Infection/etiology ; Cross Infection/microbiology ; Hematopoietic Stem Cell Transplantation/adverse effects ; Humans ; Male ; Osteosarcoma/surgery ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/therapy ; Staphylococcal Infections/drug therapy ; Staphylococcal Infections/etiology ; Staphylococcus aureus/isolation & purification
Chemical Substances	Anti-Bacterial Agents
Language	English
Publishing date	2018-01-23
Publishing country	United States
Document type	Case Reports ; Letter
ZDB-ID	639378-0
ISSN	1559-6834 ; 0195-9417 ; 0899-823X
ISSN (online)	1559-6834
ISSN	0195-9417 ; 0899-823X
DOI	10.1017/ice.2017.285
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Article ; Online: Sequential renal and bone marrow transplants in a child with Fanconi anemia.

Vincent, Carol L / Primack, William A / Hipps, John / Kasow, Kimberly A

Pediatric transplantation

2016 Volume 20, Issue 1, Page(s) 146–150

Abstract: FA is an autosomal recessive disorder characterized by small stature and renal abnormalities. FA can lead to progressive bone marrow failure, myelodysplastic syndrome, or acute leukemia. Using a multidisciplinary team approach, we managed a 3-yr-old boy ... ...

Abstract	FA is an autosomal recessive disorder characterized by small stature and renal abnormalities. FA can lead to progressive bone marrow failure, myelodysplastic syndrome, or acute leukemia. Using a multidisciplinary team approach, we managed a 3-yr-old boy with FA who simultaneously developed renal and hematopoietic failure. Because renal function was insufficient to support the conditioning regimen for HCT, we performed a deceased donor renal transplant in December 2012 prior to HCT with the known risk of graft-versus-graft rejection of the donor kidney. Seven months later he underwent allogeneic HCT. He obtained myeloid engraftment on day +11 and peripheral blood chimerism demonstrated all donor by day +21. He developed asymptomatic CMV reactivation and despite antirejection medications, mild skin graft-versus-host disease. He has maintained excellent renal function and remains transfusion independent with full hematopoietic recovery. He has not experienced any renal rejection episodes nor developed donor-specific antibodies toward his renal donor. Peripheral blood chimerism remains completely HCT donor. He is clinically well, now greater than two and a half yr after renal transplant and two yr after HCT. The continuing close collaboration between the Pediatric Nephrology and Bone Marrow Transplant teams is a major factor in this successful outcome.
MeSH term(s)	Bone Marrow Transplantation ; Child, Preschool ; Fanconi Anemia/complications ; Fanconi Anemia/surgery ; Graft Rejection ; Graft Survival/immunology ; Hematologic Diseases/complications ; Hematologic Diseases/surgery ; Hematopoietic Stem Cell Transplantation ; Humans ; Interdisciplinary Communication ; Kidney Transplantation ; Male ; Renal Insufficiency/complications ; Renal Insufficiency/surgery ; Transplantation Conditioning ; Transplantation, Homologous
Language	English
Publishing date	2016-02
Publishing country	Denmark
Document type	Case Reports ; Journal Article
ZDB-ID	1390284-2
ISSN	1399-3046 ; 1397-3142
ISSN (online)	1399-3046
ISSN	1397-3142
DOI	10.1111/petr.12619
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Zs.A 4947: Show issues

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Article ; Online: Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.

Kanter, Julie / Walters, Mark C / Krishnamurti, Lakshmanan / Mapara, Markus Y / Kwiatkowski, Janet L / Rifkin-Zenenberg, Stacey / Aygun, Banu / Kasow, Kimberly A / Pierciey, Francis J / Bonner, Melissa / Miller, Alex / Zhang, Xinyan / Lynch, Jessie / Kim, Dennis / Ribeil, Jean-Antoine / Asmal, Mohammed / Goyal, Sunita / Thompson, Alexis A / Tisdale, John F

The New England journal of medicine

2021 Volume 386, Issue 7, Page(s) 617–628

Abstract: Background: Sickle cell disease is characterized by the painful recurrence of vaso-occlusive events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; lovotibeglogene autotemcel) consists of autologous transplantation of ... ...

Abstract	Background: Sickle cell disease is characterized by the painful recurrence of vaso-occlusive events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; lovotibeglogene autotemcel) consists of autologous transplantation of hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β-globin gene, which produces an antisickling hemoglobin, HbA Methods: In this ongoing phase 1-2 study, we optimized the treatment process in the initial 7 patients in Group A and 2 patients in Group B with sickle cell disease. Group C was established for the pivotal evaluation of LentiGlobin for sickle cell disease, and we adopted a more stringent inclusion criterion that required a minimum of four severe vaso-occlusive events in the 24 months before enrollment. In this unprespecified interim analysis, we evaluated the safety and efficacy of LentiGlobin in 35 patients enrolled in Group C. Included in this analysis was the number of severe vaso-occlusive events after LentiGlobin infusion among patients with at least four vaso-occlusive events in the 24 months before enrollment and with at least 6 months of follow-up. Results: As of February 2021, cell collection had been initiated in 43 patients in Group C; 35 received a LentiGlobin infusion, with a median follow-up of 17.3 months (range, 3.7 to 37.6). Engraftment occurred in all 35 patients. The median total hemoglobin level increased from 8.5 g per deciliter at baseline to 11 g or more per deciliter from 6 months through 36 months after infusion. HbA Conclusions: One-time treatment with LentiGlobin resulted in sustained production of HbA
MeSH term(s)	Adolescent ; Adult ; Anemia, Sickle Cell/blood ; Anemia, Sickle Cell/complications ; Anemia, Sickle Cell/therapy ; Child ; Female ; Fetal Hemoglobin ; Genetic Therapy ; Genetic Vectors ; Hematopoietic Stem Cell Transplantation ; Hemoglobins/analysis ; Hemoglobins/genetics ; Hemoglobins/metabolism ; Humans ; Lentivirus ; Male ; Middle Aged ; Stem Cell Transplantation ; Vascular Patency ; Young Adult ; beta-Globins/genetics
Chemical Substances	Hemoglobins ; beta-Globins ; Fetal Hemoglobin (9034-63-3)
Language	English
Publishing date	2021-12-12
Publishing country	United States
Document type	Clinical Trial, Phase I ; Clinical Trial, Phase II ; Journal Article ; Multicenter Study ; Research Support, Non-U.S. Gov't
ZDB-ID	207154-x
ISSN	1533-4406 ; 0028-4793
ISSN (online)	1533-4406
ISSN	0028-4793
DOI	10.1056/NEJMoa2117175
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