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Article ; Online: Outcomes Using Narrowband UVB Phototherapy for Acute Cutaneous Graft Versus Host Disease in Pediatric Allogeneic Hematopoietic Stem Cell Transplant Recipients.

Snyder, Alan N / Wine Lee, Lara / Jaroscak, Jennifer J / Donahoo, Lori B / Hudspeth, Michelle

Journal of pediatric hematology/oncology

2020 Volume 43, Issue 1, Page(s) e151–e153

MeSH term(s)	Acute Disease ; Adolescent ; Adult ; Child ; Child, Preschool ; Female ; Follow-Up Studies ; Graft vs Host Disease/etiology ; Graft vs Host Disease/pathology ; Graft vs Host Disease/radiotherapy ; Hematologic Neoplasms/pathology ; Hematologic Neoplasms/therapy ; Hematopoietic Stem Cell Transplantation/adverse effects ; Humans ; Infant ; Male ; Phototherapy/methods ; Prognosis ; Retrospective Studies ; Skin Diseases/etiology ; Skin Diseases/pathology ; Skin Diseases/radiotherapy ; Transplantation, Homologous ; Ultraviolet Therapy/methods ; Young Adult
Language	English
Publishing date	2020-12-19
Publishing country	United States
Document type	Letter
ZDB-ID	1231152-2
ISSN	1536-3678 ; 1077-4114 ; 0192-8562
ISSN (online)	1536-3678
ISSN	1077-4114 ; 0192-8562
DOI	10.1097/MPH.0000000000001945
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Zs.A 1537: Show issues

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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: Anakinra utilization in refractory pediatric CAR T-cell associated toxicities.

Diorio, Caroline / Vatsayan, Anant / Talleur, Aimee C / Annesley, Colleen / Jaroscak, Jennifer J / Shalabi, Haneen / Ombrello, Amanda K / Hudspeth, Michelle / Maude, Shannon L / Gardner, Rebecca A / Shah, Nirali N

Blood advances

2022 Volume 6, Issue 11, Page(s) 3398–3403

MeSH term(s)	Child ; Humans ; Immunotherapy, Adoptive ; Interleukin 1 Receptor Antagonist Protein/therapeutic use ; Receptors, Chimeric Antigen ; T-Lymphocytes
Chemical Substances	Interleukin 1 Receptor Antagonist Protein ; Receptors, Chimeric Antigen
Language	English
Publishing date	2022-04-08
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Research Support, N.I.H., Intramural
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2022006983
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Zs.A 7153: Show issues

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Article ; Online: Apparent coexistence of ETV6::RUNX1 and KMT2A::MLLT3 fusions due to a nonproductive KMT2A rearrangement in B-ALL.

Gagnon, Marie-France / Smadbeck, James B / Sharma, Neeraj / Blackburn, Patrick R / Demasi Benevides, Jonna / Akkari, Yassmine M N / Jaroscak, Jennifer J / Znoyko, Iya / Wolff, Daynna J / Schandl, Cynthia A / Meyer, Reid / Greipp, Patricia T / Xu, Xinjie / Hoppman, Nicole L / Ketterling, Rhett P / Peterson, Jess F / Baughn, Linda B

Leukemia & lymphoma

2022 Volume 63, Issue 9, Page(s) 2243–2246

MeSH term(s)	Core Binding Factor Alpha 2 Subunit/genetics ; Humans ; Nuclear Proteins ; Oncogene Proteins, Fusion/genetics ; Precursor Cell Lymphoblastic Leukemia-Lymphoma
Chemical Substances	Core Binding Factor Alpha 2 Subunit ; MLLT3 protein, human ; Nuclear Proteins ; Oncogene Proteins, Fusion ; RUNX1 protein, human
Language	English
Publishing date	2022-05-04
Publishing country	United States
Document type	Letter ; Research Support, Non-U.S. Gov't
ZDB-ID	1042374-6
ISSN	1029-2403 ; 1042-8194
ISSN (online)	1029-2403
ISSN	1042-8194
DOI	10.1080/10428194.2022.2064991
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Zs.A 2997: Show issues

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Article ; Online: The effect of bone marrow graft composition on pediatric bone marrow transplantation outcomes.

Fabrizio, Vanessa / Wahlquist, Amy / Hill, Elise / Williams, Elizabeth / Kramer, Cindy / Jaroscak, Jennifer / Duong, Angie / Garrett-Mayer, Elizabeth / Hudspeth, Michelle

Pediatric transplantation

2018 Volume 22, Issue 8, Page(s) e13287

Abstract: Hematopoietic stem cell graft cellular composition has been generally accepted to impact outcomes. Recent studies question this hypothesis. We conducted a single-center retrospective study of sixty-one pediatric BMT recipients for malignant (68%) and ... ...

Abstract	Hematopoietic stem cell graft cellular composition has been generally accepted to impact outcomes. Recent studies question this hypothesis. We conducted a single-center retrospective study of sixty-one pediatric BMT recipients for malignant (68%) and nonmalignant diseases (32%) examining effects of graft composition on engraftment, acute GVHD, chronic GVHD, and survival at day 100 and 1 year. Grafts contained a median of 3.63 x 0
MeSH term(s)	Adolescent ; Antigens, CD34/metabolism ; Blood Platelets/cytology ; Bone Marrow/pathology ; Bone Marrow Transplantation ; Child ; Child, Preschool ; Female ; Graft Survival ; Graft vs Host Disease/etiology ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/cytology ; Humans ; Infant ; Male ; Neoplasms/therapy ; Neutrophils/cytology ; Reproducibility of Results ; Retrospective Studies ; Transplantation, Homologous ; Treatment Outcome ; Young Adult
Chemical Substances	Antigens, CD34
Language	English
Publishing date	2018-08-29
Publishing country	Denmark
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	1390284-2
ISSN	1399-3046 ; 1397-3142
ISSN (online)	1399-3046
ISSN	1397-3142
DOI	10.1111/petr.13287
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Zs.A 4947: Show issues

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Article ; Online: Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.

Goyal, Sunita / Tisdale, John / Schmidt, Manfred / Kanter, Julie / Jaroscak, Jennifer / Whitney, Dustin / Bitter, Hans / Gregory, Philip D / Parsons, Geoffrey / Foos, Marianna / Yeri, Ashish / Gioia, Maple / Voytek, Sarah B / Miller, Alex / Lynch, Jessie / Colvin, Richard A / Bonner, Melissa

The New England journal of medicine

2021 Volume 386, Issue 2, Page(s) 138–147

Abstract: Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel) consists of autologous transplantation of a patient's hematopoietic stem cells transduced with the BB305 lentiviral vector that encodes the ... ...

Abstract	Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel) consists of autologous transplantation of a patient's hematopoietic stem cells transduced with the BB305 lentiviral vector that encodes the β
MeSH term(s)	Adult ; Anemia, Sickle Cell/complications ; Anemia, Sickle Cell/genetics ; Anemia, Sickle Cell/therapy ; Carcinogenesis ; Female ; Gene Expression ; Genetic Therapy/adverse effects ; Genetic Vectors ; Hematopoietic Stem Cell Transplantation ; Humans ; Lentivirus ; Leukemia, Myeloid, Acute/etiology ; Risk Factors ; Sequence Analysis, RNA ; Transgenes ; Transplantation, Autologous ; beta-Globins/genetics
Chemical Substances	beta(A-T87Q) globin ; beta-Globins
Language	English
Publishing date	2021-12-12
Publishing country	United States
Document type	Case Reports ; Journal Article ; 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/NEJMoa2109167
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Ua VI Zs.34: Show issues

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Article ; Online: Alemtuzumab based reduced intensity transplantation for pediatric severe aplastic anemia.

Ngwube, Alexander / Hayashi, Robert J / Murray, Lisa / Loechelt, Brett / Dalal, Jignesh / Jaroscak, Jennifer / Shenoy, Shalini

Pediatric blood & cancer

2015 Volume 62, Issue 7, Page(s) 1270–1276

Abstract: Background: Hematopoietic cell transplantation (HCT) is curative in patients with severe aplastic anemia (SAA). HCT is considered at presentation when a HLA-matched related donor (MRD) is available and has a high success rate. Unrelated donor (URD) ... ...

Abstract	Background: Hematopoietic cell transplantation (HCT) is curative in patients with severe aplastic anemia (SAA). HCT is considered at presentation when a HLA-matched related donor (MRD) is available and has a high success rate. Unrelated donor (URD) transplants are typically undertaken if immunosuppressive therapy fails. Increased toxicity and graft rejection are often encountered in this setting. Procedure: We report a prospective multi-center trial of HCT in 17 children with SAA following novel reduced intensity conditioning with alemtuzumab, fludarabine and melphalan, and the best available donor. Nine were URD transplants matched at 7-8/8 loci, and performed following failure of immune suppression. Median follow up was 61 months (range 6-128). Results: All patients engrafted. Estimated 5 year event-free and overall-survival was 88% (95%CI 65.7-96.7). Five year overall survival for MRD and URD transplants was 100% and 78% (95%CI 45-93.6) respectively. Median times to neutrophil and platelet engraftment was 14 (range 10-27) and 23.5 (range 11-65) days respectively. Treatment related mortality was 12%. The incidence of grade II-IV and III-IV acute graft-versus-host disease was 29% and 18% respectively. At two years, all but one patient discontinued immunosuppression successfully. Laboratory measures of immune reconstitution normalized at one year and infection rates were low in the latter part of the first year. Conclusions: HCT using this RIC approach was well tolerated and successful in achieving donor engraftment and early immune reconstitution with good quality of life free of immune suppression. Children with SAA can be successfully transplanted using alemtuzumab based conditioning.
MeSH term(s)	Adolescent ; Alemtuzumab ; Anemia, Aplastic/complications ; Anemia, Aplastic/mortality ; Anemia, Aplastic/therapy ; Antibodies, Monoclonal, Humanized/administration & dosage ; Antineoplastic Agents/administration & dosage ; Child ; Child, Preschool ; Combined Modality Therapy ; Female ; Follow-Up Studies ; Graft vs Host Disease/etiology ; Graft vs Host Disease/mortality ; Hematopoietic Stem Cell Transplantation ; Humans ; Male ; Prognosis ; Prospective Studies ; Quality of Life ; Survival Rate ; Transplantation Conditioning ; Transplantation, Homologous ; Unrelated Donors
Chemical Substances	Antibodies, Monoclonal, Humanized ; Antineoplastic Agents ; Alemtuzumab (3A189DH42V)
Language	English
Publishing date	2015-07
Publishing country	United States
Document type	Clinical Trial, Phase II ; Journal Article ; Multicenter Study
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.25458
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Zs.A 1131: Show issues

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Article ; Online: Immune Reconstitution and Infection Patterns after Early Alemtuzumab and Reduced Intensity Transplantation for Nonmalignant Disorders in Pediatric Patients.

Bhatt, Sima T / Bednarski, Jeffrey J / Berg, Julia / Trinkaus, Kathryn / Murray, Lisa / Hayashi, Robert / Schulz, Ginny / Hente, Monica / Grimley, Michael / Chan, Ka Wah / Kamani, Naynesh / Jacobsohn, David / Nieder, Michael / Hale, Gregory / Yu, Lolie / Adams, Roberta / Dalal, Jignesh / Pulsipher, Michael A / Haut, Paul /

Chaudhury, Sonali / Davis, Jeffrey / Jaroscak, Jennifer / Andreansky, Martin / Willert, Jennifer / Shenoy, Shalini

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

2018 Volume 25, Issue 3, Page(s) 556–561

Abstract: Hematopoietic stem cell transplantation (HSCT) is a therapeutic option for many nonmalignant disorders (NMD) and is curative or prevents disease progression. Reduced-intensity conditioning (RIC) in HSCT for NMD may reduce regimen-related acute toxicities ...

Abstract	Hematopoietic stem cell transplantation (HSCT) is a therapeutic option for many nonmalignant disorders (NMD) and is curative or prevents disease progression. Reduced-intensity conditioning (RIC) in HSCT for NMD may reduce regimen-related acute toxicities and late complications. Myeloablation is often replaced by immune suppression in RIC regimens to support donor engraftment. The pace of immune reconstitution after immune suppression by RIC regimens is influenced by agents used, donor source, and graft-versus-host disease prophylaxis/treatment. In a multicenter trial (NCT 00920972) of HSCT for NMD, a RIC regimen consisting of alemtuzumab, fludarabine, and melphalan was substituted for myeloablation. Alemtuzumab was administered early (days -21 to -19) to mitigate major lymphodepletion of the incoming graft and the risk of graft rejection. Immune reconstitution and infectious complications were prospectively monitored for 1-year post-HSCT. Seventy-one patients met inclusion criteria for this report and received marrow or peripheral blood stem cell transplants. Immune reconstitution and infections are reported for related donor (RD) and unrelated donor (URD) transplants at 3 time-points (100days, 6 months, and 1 year post-HSCT). Natural killer cell recovery was rapid, and numbers normalized in both cohorts by day +100. Mean CD3, CD4, and CD8 T-lymphocyte numbers normalized by 6 months after RD HSCT and by 1 year in the URD group. CD4 and CD8 T-lymphocyte counts were significantly higher in patients who received RD HSCT at 6 months and at 1 year, respectively, post-HSCT compared with patients who received URD HSCT. The pace of CD19 B-cell recovery was markedly different between RD and URD cohorts. Mean B-cell numbers were normal by day 100 after RD HSCT but took 1 year post-HSCT to normalize in the URD cohort. Despite these differences in immune reconstitution, the timing and nature of infections did not differ between the groups, presumably because of comparable T-lymphocyte recovery. Immune reconstitution occurred at a faster pace than in prior reports using RIC with T-cell depletion. The incidence of infections was similar for both cohorts and occurred most frequently in the first 100days post-HSCT. Viral and fungal infections occurred at a lower incidence in this cohort, with "early" alemtuzumab compared with regimens administering serotherapy in the peritransplantation period. Patients were susceptible to bacterial infections primarily in the first 100days irrespective of donor source and had no increase in mortality from the same. The overall mortality rate from infections was 1.4% at 1 year. Close monitoring and prophylaxis against bacterial infections in the first 100days post-HSCT is necessary but is followed by robust immune reconstitution, especially in the T-cell compartment.
MeSH term(s)	Alemtuzumab/adverse effects ; Alemtuzumab/therapeutic use ; Child ; Female ; Graft Survival ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Immune Reconstitution ; Infections/etiology ; Lymphocyte Depletion ; Male ; Survival Analysis ; Transplantation Conditioning/methods ; Unrelated Donors
Chemical Substances	Alemtuzumab (3A189DH42V)
Language	English
Publishing date	2018-10-12
Publishing country	United States
Document type	Clinical Trial ; Journal Article ; Multicenter Study ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	1474865-4
ISSN	1523-6536 ; 1083-8791
ISSN (online)	1523-6536
ISSN	1083-8791
DOI	10.1016/j.bbmt.2018.10.008
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Zs.A 5082: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
Zs.MO 462: Show issues

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Article ; Online: Successful treatment of EBV-associated posttransplantation lymphoma after cord blood transplantation using third-party EBV-specific cytotoxic T lymphocytes.

Barker, Juliet N / Doubrovina, Ekaterina / Sauter, Craig / Jaroscak, Jennifer J / Perales, Miguel A / Doubrovin, Mikhail / Prockop, Susan E / Koehne, Guenther / O'Reilly, Richard J

Blood

2010 Volume 116, Issue 23, Page(s) 5045–5049

Abstract: Cellular therapy of Epstein-Barr virus (EBV)(+) posttransplantation lymphoproliferative diseases (PTLD) in cord blood transplant (CBT) recipients is limited by lack of donor access and the donor's naive neonatal immune system. We therefore used partially ...

Abstract	Cellular therapy of Epstein-Barr virus (EBV)(+) posttransplantation lymphoproliferative diseases (PTLD) in cord blood transplant (CBT) recipients is limited by lack of donor access and the donor's naive neonatal immune system. We therefore used partially human leukocyte antigen-matched third-party in vitro expanded EBV-specific cytotoxic T lymphocytes (CTLs) to treat 2 CBT recipients with life-threatening, donor-derived monoclonal EBV(+) diffuse large B-cell lymphomas with extranodal involvement developing in the context of graft-versus-host disease. Both patients had failed immunosuppression taper and Rituximab. After 5 and 9 infusions of 10(6) EBV-CTL/kg, respectively, each patient achieved a sustained complete remission without toxicity or graft-versus-host disease. Each is alive without recurrence at 20 and 15 months, respectively, post-EBV-PTLD diagnosis. This approach demonstrates the efficacy of using "off-the-shelf," virus-specific third-party CTLs restricted by human leukocyte antigens expressed by the tumor to treat otherwise lethal EBV-PTLD. Such therapy may also be applicable to the treatment of other infections and residual or recurrent malignancy after CBT.
MeSH term(s)	Adult ; Child ; Cord Blood Stem Cell Transplantation/adverse effects ; Epstein-Barr Virus Infections/complications ; Female ; Graft vs Host Disease/etiology ; Graft vs Host Disease/immunology ; Graft vs Host Disease/therapy ; HLA Antigens/genetics ; HLA Antigens/immunology ; Humans ; Immunotherapy/methods ; Lymphoma/surgery ; Lymphoma, Large B-Cell, Diffuse/therapy ; Lymphoma, Large B-Cell, Diffuse/virology ; Male ; T-Lymphocytes, Cytotoxic/immunology ; T-Lymphocytes, Cytotoxic/transplantation
Chemical Substances	HLA Antigens
Language	English
Publishing date	2010-09-08
Publishing country	United States
Document type	Case Reports ; Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood-2010-04-281873
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Article ; Online: A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.

Shenoy, Shalini / Eapen, Mary / Panepinto, Julie A / Logan, Brent R / Wu, Juan / Abraham, Allistair / Brochstein, Joel / Chaudhury, Sonali / Godder, Kamar / Haight, Ann E / Kasow, Kimberly A / Leung, Kathryn / Andreansky, Martin / Bhatia, Monica / Dalal, Jignesh / Haines, Hilary / Jaroscak, Jennifer / Lazarus, Hillard M / Levine, John E /

Krishnamurti, Lakshmanan / Margolis, David / Megason, Gail C / Yu, Lolie C / Pulsipher, Michael A / Gersten, Iris / DiFronzo, Nancy / Horowitz, Mary M / Walters, Mark C / Kamani, Naynesh

Blood

2016 Volume 128, Issue 21, Page(s) 2561–2567

Abstract: Children with sickle cell disease experience organ damage, impaired quality of life, and premature mortality. Allogeneic bone marrow transplant from an HLA-matched sibling can halt disease progression but is limited by donor availability. A Blood and ... ...

Abstract	Children with sickle cell disease experience organ damage, impaired quality of life, and premature mortality. Allogeneic bone marrow transplant from an HLA-matched sibling can halt disease progression but is limited by donor availability. A Blood and Marrow Transplant Clinical Trials Network (BMT CTN) phase 2 trial conducted from 2008 to 2014 enrolled 30 children aged 4 to 19 years; 29 were eligible for evaluation. The primary objective was 1-year event-free survival (EFS) after HLA allele-matched (at HLA-A, -B, -C, and -DRB1 loci) unrelated donor transplant. The conditioning regimen included alemtuzumab, fludarabine, and melphalan. Graft-versus-host disease (GVHD) prophylaxis included calcineurin inhibitor, short-course methotrexate, and methylprednisolone. Transplant indications included stroke (n = 12), transcranial Doppler velocity >200 cm/s (n = 2), ≥3 vaso-occlusive pain crises per year (n = 12), or ≥2 acute chest syndrome episodes (n = 4) in the 2 years preceding enrollment. Median follow-up was 26 months (range, 12-62 months); graft rejection was 10%. The 1- and 2-year EFS rates were 76% and 69%, respectively. The corresponding rates for overall survival were 86% and 79%. The day 100 incidence rate of grade II-IV acute GVHD was 28%, and the 1-year incidence rate of chronic GVHD was 62%; 38% classified as extensive. There were 7 GVHD-related deaths. A 34% incidence of posterior reversible encephalopathy syndrome was noted in the first 6 months. Although the 1-year EFS met the prespecified target of ≥75%, this regimen cannot be considered sufficiently safe for widespread adoption without modifications to achieve more effective GVHD prophylaxis. The BMT CTN #0601 trial was registered at www.clinicaltrials.gov as #NCT00745420.
MeSH term(s)	Adolescent ; Allografts ; Anemia, Sickle Cell/mortality ; Anemia, Sickle Cell/physiopathology ; Anemia, Sickle Cell/therapy ; Bone Marrow Transplantation ; Calcineurin Inhibitors/administration & dosage ; Child ; Disease-Free Survival ; Female ; Follow-Up Studies ; Graft vs Host Disease/mortality ; Graft vs Host Disease/prevention & control ; Humans ; Male ; Survival Rate ; Unrelated Donors
Chemical Substances	Calcineurin Inhibitors
Language	English
Publishing date	2016-09-13
Publishing country	United States
Document type	Clinical Trial ; Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood-2016-05-715870
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