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Article ; Online: Liver abnormalities are frequent and persistent in patients with Fanconi anemia.

Snyder, Alana J / Campbell, Kathleen M / Lane, Adam / Mehta, Parinda A / Myers, Kasiani / Davies, Stella M / Koo, Jane

2024 Volume 8, Issue 6, Page(s) 1427–1438

Abstract: Abstract: Liver disease has not been well described in patients with Fanconi anemia (FA). Improvements in outcomes of transplant mean that more individuals with FA are reaching adulthood and new features of the FA phenotype are being discovered. We ... ...

Abstract	Abstract: Liver disease has not been well described in patients with Fanconi anemia (FA). Improvements in outcomes of transplant mean that more individuals with FA are reaching adulthood and new features of the FA phenotype are being discovered. We performed a retrospective review of liver function in a cohort of 97 patients with FA followed-up for at least 10 years at a single center. We identified a high frequency of transaminitis (n = 31, 32%) without elevation of bilirubin and with no evidence of structural hepatic abnormality in patients with FA. Transaminitis was persistent in many cases, sometimes lasting more than a decade without clinical manifestation, although 2 patients with prolonged transaminitis are deceased from liver failure, indicating important long-term clinical consequences. Transaminitis was found in patients who had and had not received transplant but was more frequent in recipients of transplant. Exposure to total body irradiation increased risk (odds ratio, 15.5 [95% confidence interval, 2.44-304.54]; P = .01), whereas treatment with androgens did not. Review of limited numbers of liver biopsies and autopsy material showed a cholestatic pattern of liver injury, with progressive fibrosis, in the majority of patients. Occurrence in cases without transplant as well as cases with transplant argues against a potential diagnosis of atypical liver graft-versus-host disease. Limited data regarding therapy suggest no benefit from treatment with steroids or other immune suppressive medications or ursodeoxycholic acid. Our data show that liver disease is common in patients with FA, and because most children with FA now reach adulthood, end-stage liver disease in young adulthood means systematic testing of potential therapies is urgently needed.
MeSH term(s)	Child ; Humans ; Young Adult ; Adult ; Fanconi Anemia/complications ; Fanconi Anemia/therapy ; Liver Diseases ; Androgens/adverse effects ; Phenotype
Chemical Substances	Androgens
Language	English
Publishing date	2024-01-17
Publishing country	United States
Document type	Journal Article
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2023012215
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Zs.A 7153: Show issues

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Article ; Online: Gonadal function in pediatric Fanconi anemia patients treated with hematopoietic stem cell transplant.

Koo, Jane / Grom-Mansencal, Ines / Howell, Jonathan C / Rios, Julie M / Mehta, Parinda A / Davies, Stella M / Myers, Kasiani C

Haematologica

2023 Volume 108, Issue 9, Page(s) 2358–2368

Abstract: Gonadal dysfunction and reduced fertility are clinical manifestations well described in patients with Fanconi anemia (FA) and following hematopoietic stem cell transplantation (HSCT). It is difficult to differentiate gonadal dysfunction from the primary ... ...

Abstract	Gonadal dysfunction and reduced fertility are clinical manifestations well described in patients with Fanconi anemia (FA) and following hematopoietic stem cell transplantation (HSCT). It is difficult to differentiate gonadal dysfunction from the primary disease itself or from HSCT procedures. Therefore, it is important to manage expectations about gonadal failure and infertility for all patients with FA, regardless of the HSCT status. We performed a retrospective analysis of 98 pediatric patients with FA who were transplanted between July 1990 and June 2020 to evaluate the incidence of gonadal dysfunction in female and male patients with FA. New-onset premature ovarian insufficiency (POI) was diagnosed in a total of 30 (52.6%) patients. Follicle-stimulating hormone and luteinizing hormone levels were increased in patients diagnosed with POI. Anti- Mullerian hormone levels declined in POI patients after HSCT (r2=0.21; P=0.001). Twenty (48.8%) male patients were diagnosed with testicular failure. Follicle-stimulating hormone levels increased after HSCT even in patients without testicular failure (r2=0.17; P=0.005). Inhibin B levels decreased over time after HSCT in patients with testicular failure (r2=0.14; P=0.001). These data indicate brisk decline in already impaired gonadal function in transplanted children with FA.
MeSH term(s)	Humans ; Child ; Male ; Female ; Retrospective Studies ; Fanconi Anemia/complications ; Fanconi Anemia/therapy ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cell Transplantation/methods ; Follicle Stimulating Hormone ; Primary Ovarian Insufficiency/etiology ; Primary Ovarian Insufficiency/diagnosis
Chemical Substances	Follicle Stimulating Hormone (9002-68-0)
Language	English
Publishing date	2023-09-01
Publishing country	Italy
Document type	Journal Article
ZDB-ID	2333-4
ISSN	1592-8721 ; 0017-6567 ; 0390-6078
ISSN (online)	1592-8721
ISSN	0017-6567 ; 0390-6078
DOI	10.3324/haematol.2022.282094
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Article ; Online: Ruxolitinib Pharmacokinetics and Pharmacodynamics in Children with Acute and Chronic Graft-versus-Host Disease.

Cook, Eleanor / Dong, Min / Chiang, Samuel C C / Luedeke, David / Lake, Kelly E / Hoerth, Colin / Deavy, Marisa / Setchell, Kenneth D R / Zhao, Junfang / Punt, Nieko / Galletta, Thomas / Teusink-Cross, Ashley / Davies, Stella M / Marsh, Rebecca A / Mehta, Parinda / Khandelwal, Pooja

Transplantation and cellular therapy

2024 Volume 30, Issue 5, Page(s) 528.e1–528.e12

Abstract: We evaluated the pharmacokinetics (PK) of oral ruxolitinib in children with steroid-refractory acute graft-versus-host disease (aGVHD) (age <12 years) and chronic GVHD (cGVHD) (age ≤18 years) using our published pediatric dosing. PK sampling was ... ...

Abstract	We evaluated the pharmacokinetics (PK) of oral ruxolitinib in children with steroid-refractory acute graft-versus-host disease (aGVHD) (age <12 years) and chronic GVHD (cGVHD) (age ≤18 years) using our published pediatric dosing. PK sampling was performed before and 2 hours after ruxolitinib administration in patients with established cGVHD. More extensive PK analyses were performed in patients with newly diagnosed aGVHD or cGVHD before and .5, 1, 2, 4, and 6 hours after ruxolitinib administration in patients weighing >10 kg and before, 3+, and 6+ hours in children weighing <10 kg. pSTAT1, pSTAT3, and pSTAT5 expression levels were measured on CD4
MeSH term(s)	Humans ; Graft vs Host Disease/drug therapy ; Pyrazoles/pharmacokinetics ; Pyrazoles/therapeutic use ; Child ; Nitriles ; Pyrimidines/pharmacokinetics ; Pyrimidines/therapeutic use ; Child, Preschool ; Male ; Female ; Chronic Disease ; Adolescent ; Infant ; Acute Disease ; STAT1 Transcription Factor/metabolism ; STAT3 Transcription Factor/metabolism ; STAT5 Transcription Factor/metabolism ; Prospective Studies ; Hematopoietic Stem Cell Transplantation ; Bronchiolitis Obliterans Syndrome
Chemical Substances	ruxolitinib (82S8X8XX8H) ; Pyrazoles ; Nitriles ; Pyrimidines ; STAT1 Transcription Factor ; STAT3 Transcription Factor ; STAT5 Transcription Factor ; STAT1 protein, human ; STAT3 protein, human
Language	English
Publishing date	2024-02-22
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	3062231-1
ISSN	2666-6367
ISSN (online)	2666-6367
DOI	10.1016/j.jtct.2024.02.018
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Zs.A 5082: Show issues

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Article: Paperspray Ionization Mass Spectrometry as a Tool for Predicting Real-Time Optimized Dosing of the Chemotherapeutic Drug Melphalan.

Zhao, Junfang / Sharat, Chandra / Mehta, Parinda A / Mizuno, Kana / Vinks, Alexander A / Setchell, Kenneth D R

The journal of applied laboratory medicine

2021 Volume 6, Issue 3, Page(s) 625–636

Abstract: Background: Melphalan, an important component of hematopoietic stem cell transplantation (HSCT) preparative regimens, is associated with significant toxicity and large between patient variability in pharmacokinetics making it difficult to calculate the ... ...

Abstract	Background: Melphalan, an important component of hematopoietic stem cell transplantation (HSCT) preparative regimens, is associated with significant toxicity and large between patient variability in pharmacokinetics making it difficult to calculate the optimal dose for pediatric patients. Paperspray (PS) ionization generates gas phase analyte ions directly from a dried blood spot without the need for prior sample preparation or chromatography. With these advantages, a validated PS-MS/MS assay was developed and applied to the 'real-time' determination of melphalan pharmacokinetics (PK). Methods: Melphalan was quantified by stable-labeled isotope dilution analysis in whole blood by PS-MS/MS. Blood samples were obtained at timed intervals from patients during HSCT after administration of a very low (test) dose of melphalan to avoid toxicity. Pharmacokinetics parameters were calculated using WinNonlin v.6.4. From these data, the optimal therapeutic dose was estimated and full dose PK repeated. Results: PS-MS/MS method was linear over a large dynamic range (25-50 000 ng/mL), intra- and interassay reproducibility of quality control samples was <15% CV. With essentially no prior sample preparation, PS-MS/MS measurement of blood melphalan concentrations showed excellent correlation (R2 = 0.959, n = 62) with a validated electrospray-LC-MS/MS method. Trapezoidal area under the curves calculated for 5 patients administered low dose melphalan showed a high linear correlation (R2 = 0.981) between the PS-MS/MS and LC-MS/MS methods. The faster PS approach permitted real-time PK evaluation of individual patients. Conclusions: A validated PS-MS/MS assay for melphalan in patients undergoing HSCT is described that facilitates pharmacokinetic-guided individualized precision dosing with immediate bedside dose adjustments to improve outcomes by balancing toxicity and efficacy of melphalan.
MeSH term(s)	Child ; Chromatography, Liquid ; Humans ; Melphalan ; Pharmaceutical Preparations ; Reproducibility of Results ; Tandem Mass Spectrometry
Chemical Substances	Pharmaceutical Preparations ; Melphalan (Q41OR9510P)
Language	English
Publishing date	2021-02-11
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ISSN	2576-9456
ISSN	2576-9456
DOI	10.1093/jalm/jfaa237
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Article ; Online: Phenotypes of adults with Fanconi anaemia.

Wang, YunZu Michele / Loveless, Michaela / Miller, Erica / Nelson, Adam S / Mehta, Parinda A / Davies, Stella M / Myers, Kasiani C

British journal of haematology

2022 Volume 201, Issue 1, Page(s) 133–139

Abstract: The long-term outcomes of adults with Fanconi anaemia (FA) have improved with advances in haematopoietic stem cell transplantation (HSCT) and more detailed follow-up and screening guidelines. The phenotype of those who survive to adulthood may differ ... ...

Abstract	The long-term outcomes of adults with Fanconi anaemia (FA) have improved with advances in haematopoietic stem cell transplantation (HSCT) and more detailed follow-up and screening guidelines. The phenotype of those who survive to adulthood may differ from the typical presentation of FA. We collected retrospective clinical data on adults with FA who received their care at the Cincinnati Children's Hospital Medical Center. In our final cohort of 52 patients, there were 29 females and 23 males, with median (range) age of 21 (18-37) years. Overall, 42 patients (81%) were alive at last follow-up. In all, 36 adults (69%) had undergone HSCT, including eight who had developed myelodysplasia or acute myeloid leukaemia. Eight (15%) developed squamous cell carcinoma. Endocrine complications were common, including hypothyroidism (42%), diabetes (10%), low body mass index (31%) and low bone mineral density (51%). The majority of adults with FA were employed (52%) or full-time students (13%). A significant subset of patients with FA are surviving into adulthood without requiring HSCT. Endocrine abnormalities and the development of solid tumours complicate adulthood. With improved survival outcomes following HSCT and more aggressive malignancy screening protocols, ongoing longitudinal analysis will be important to further characterise this cohort and the phenotype of untransplanted adults with FA.
MeSH term(s)	Male ; Female ; Humans ; Fanconi Anemia/complications ; Fanconi Anemia/diagnosis ; Fanconi Anemia/therapy ; Retrospective Studies ; Hematopoietic Stem Cell Transplantation/methods ; Myelodysplastic Syndromes/complications ; Phenotype
Language	English
Publishing date	2022-12-13
Publishing country	England
Document type	Journal Article
ZDB-ID	80077-6
ISSN	1365-2141 ; 0007-1048
ISSN (online)	1365-2141
ISSN	0007-1048
DOI	10.1111/bjh.18603
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Article ; Online: Unique Challenges of Hematopoietic Cell Transplantation in Adolescent and Young Adults with Hematologic Malignancies.

Mehta, Parinda A / Rotz, Seth J / Majhail, Navneet S

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

2018 Volume 24, Issue 12, Page(s) e11–e19

Abstract: Slow but definite progress has been made in recognizing the differences in disease biology, host factors, and psychosocial issues related to management of hematologic malignancies, including hematopoietic cell transplantation (HCT), in the adolescent and ...

Abstract	Slow but definite progress has been made in recognizing the differences in disease biology, host factors, and psychosocial issues related to management of hematologic malignancies, including hematopoietic cell transplantation (HCT), in the adolescent and young adult (AYA) population. Improvement in survival rates among AYAs undergoing HCT for hematologic malignancies have paralleled those seen among children and older adults in the contemporary era. However, overall outcomes remain inferior compared with those in children owing largely to higher rates of treatment related mortality and late relapses in AYA HCT recipients. We review the unique issues and challenges facing AYAs with hematologic malignancies undergoing HCT, identify unmet needs and gaps in care, and describe potential strategies to address them. A patient-centric and multidisciplinary approach to AYA HCT care with lifelong follow-up is necessary to ensure long-term health.
MeSH term(s)	Adolescent ; Adult ; Female ; Hematologic Neoplasms/pathology ; Hematologic Neoplasms/therapy ; Hematopoietic Stem Cell Transplantation/methods ; Humans ; Male ; Young Adult
Language	English
Publishing date	2018-09-19
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	1474865-4
ISSN	1523-6536 ; 1083-8791
ISSN (online)	1523-6536
ISSN	1083-8791
DOI	10.1016/j.bbmt.2018.09.011
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Article ; Online: Model-informed precision dosing for alemtuzumab in paediatric and young adult patients undergoing allogeneic haematopoietic cell transplantation.

Dong, Min / Emoto, Chie / Fukuda, Tsuyoshi / Arnold, Danielle E / Mehta, Parinda A / Marsh, Rebecca A / Vinks, Alexander A

British journal of clinical pharmacology

2021 Volume 88, Issue 1, Page(s) 248–259

Abstract: Alemtuzumab is a lymphodepleting monoclonal antibody utilized in conditioning regimens for allogeneic haematopoietic cell transplantation (HCT). A recently proposed therapeutic range of 0.15-0.6 μg/mL on the day of transplantation is associated with ... ...

Abstract	Alemtuzumab is a lymphodepleting monoclonal antibody utilized in conditioning regimens for allogeneic haematopoietic cell transplantation (HCT). A recently proposed therapeutic range of 0.15-0.6 μg/mL on the day of transplantation is associated with better HCT outcomes. The purpose of this study was to characterize alemtuzumab population pharmacokinetic/pharmacodynamic (PK/PD) and to propose individualized subcutaneous dosing schemes to achieve this optimal level for paediatric patients. Methods: Alemtuzumab concentration and absolute lymphocyte count (ALC) profiles were obtained from 29 paediatric and young adult patients (median age 6.4 y; range 0.28-21.4 y) with nonmalignant disorders undergoing HCT. PK/PD analyses were performed using nonlinear mixed effects modelling. Monte Carlo simulation was conducted to evaluate different improved dosing approaches. Results: A one-compartment model with sequential zero- and first-order absorption adequately described subcutaneously administered alemtuzumab PK. Model fit was significantly improved by including allometrically scaled body weight on clearance (0.080 L/h/70 kg) and volume of distribution (17.4 L/70 kg). ALC reduction following subcutaneous alemtuzumab was swift. An inhibitory E Conclusion: An allometry- or body surface area-based starting dosing regimen in combination with individualized Bayesian PK estimation using concentration feedback is proposed for alemtuzumab precision dosing in children undergoing allogeneic HCT.
MeSH term(s)	Alemtuzumab ; Bayes Theorem ; Child ; Computer Simulation ; Hematopoietic Stem Cell Transplantation ; Humans ; Transplantation Conditioning ; Young Adult
Chemical Substances	Alemtuzumab (3A189DH42V)
Language	English
Publishing date	2021-07-18
Publishing country	England
Document type	Journal Article ; Research Support, Non-U.S. Gov't
ZDB-ID	188974-6
ISSN	1365-2125 ; 0306-5251 ; 0264-3774
ISSN (online)	1365-2125
ISSN	0306-5251 ; 0264-3774
DOI	10.1111/bcp.14955
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Zs.A 1118: Show issues

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Article ; Online: Influence of CYP2D6 metabolizer status on ondansetron efficacy in pediatric patients undergoing hematopoietic stem cell transplantation: A case series.

Edwards, Andrea / Teusink-Cross, Ashley / Martin, Lisa J / Prows, Cynthia A / Mehta, Parinda A / Ramsey, Laura B

Clinical and translational science

2021 Volume 15, Issue 3, Page(s) 610–618

Abstract: Chemotherapy-induced nausea and vomiting (CINV) is commonly experienced by patients receiving antineoplastic agents prior to hemopoietic stem cell transplant (HSCT). Ondansetron, a 5-HT3 antagonist metabolized by CYP2D6, is an antiemetic prescribed to ... ...

Abstract	Chemotherapy-induced nausea and vomiting (CINV) is commonly experienced by patients receiving antineoplastic agents prior to hemopoietic stem cell transplant (HSCT). Ondansetron, a 5-HT3 antagonist metabolized by CYP2D6, is an antiemetic prescribed to treat short-term CINV, but some patients still experience uncontrolled nausea and vomiting while taking ondansetron. Adult CYP2D6 ultrarapid metabolizers (UMs) are at higher risk for CINV due to rapid ondansetron clearance, but similar studies have not been performed in pediatric patients. We performed a retrospective chart review of 128 pediatric HSCT recipients who received ondansetron for CINV prevention and had CYP2D6 genotyping for 20 alleles and duplication detection. The number of emetic episodes for each patient was collected from the start of chemotherapy through 7 days after HSCT. The average age of the cohort was 6.6 years (range: 0.2-16.7) and included three UMs, 72 normal metabolizers, 47 intermediate metabolizers, and six poor metabolizers. Because UMs are the population at risk for inefficacy, we describe the course of treatment for these three patients, as well as the factors influencing emesis: chemotherapy emetogenicity, diagnosis, and duration of ondansetron administration. The cases described support guidelines recommending non-CYP2D6 metabolized antiemetics (e.g., granisetron) when a patient is a known CYP2D6 UM, but pediatric studies with a larger sample of CYP2D6 UMs are needed to validate our findings.
MeSH term(s)	Adolescent ; Adult ; Antiemetics/therapeutic use ; Antineoplastic Agents/therapeutic use ; Child ; Child, Preschool ; Cytochrome P-450 CYP2D6/genetics ; Hematopoietic Stem Cell Transplantation/adverse effects ; Humans ; Infant ; Nausea/chemically induced ; Ondansetron/pharmacology ; Ondansetron/therapeutic use ; Retrospective Studies ; Vomiting/chemically induced ; Vomiting/drug therapy ; Vomiting/prevention & control
Chemical Substances	Antiemetics ; Antineoplastic Agents ; Ondansetron (4AF302ESOS) ; Cytochrome P-450 CYP2D6 (EC 1.14.14.1)
Language	English
Publishing date	2021-10-20
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural
ZDB-ID	2433157-0
ISSN	1752-8062 ; 1752-8054
ISSN (online)	1752-8062
ISSN	1752-8054
DOI	10.1111/cts.13171
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Article ; Online: T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML.

Satty, Alexandra M / Klein, Elizabeth / Mauguen, Audrey / Kunvarjee, Binni / Boelens, Jaap Jan / Cancio, Maria / Curran, Kevin J / Kernan, Nancy A / Prockop, Susan E / Scaradavou, Andromachi / Spitzer, Barbara / Tamari, Roni / Ruggiero, Julianne / Torok-Castanza, Joanne / Mehta, Parinda A / O'Reilly, Richard J / Boulad, Farid

Bone marrow transplantation

2023 Volume 59, Issue 1, Page(s) 23–33

Abstract: The only curative approach for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) arising in patients with Fanconi anemia (FA) is allogeneic hematopoietic stem cell transplantation (HCT); however, HCT approaches are inconsistent and limited ... ...

Abstract	The only curative approach for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) arising in patients with Fanconi anemia (FA) is allogeneic hematopoietic stem cell transplantation (HCT); however, HCT approaches are inconsistent and limited data on outcomes exist. We retrospectively evaluated outcomes of thirty patients with FA and MDS/AML who underwent first allogeneic HCT with a T-cell depleted (TCD) graft at our institution. Patients were transplanted on successive protocols with stepwise changes in cytoreduction and GVHD prophylaxis. All but two patients (93%) experienced durable hematopoietic engraftment. With median follow-up of 8.7 years, 5-year OS was 66.8% and DFS 53.8%. No significant differences in survival were found in patients with high-risk prognostic features (age ≥20 years, AML diagnosis, alternative donor graft) or when stratified by conditioning regimen. The 5-year cumulative incidences of relapse and NRM were 24.3% and 21.9%, respectively. NRM was higher in patients ≥20 years at HCT but did not otherwise differ. We herein demonstrate promising outcomes following allogeneic HCT for patients with FA and MDS/AML using TCD grafts, particularly in a cohort of high-risk patients with 50% ≥20 years and a majority receiving mismatched grafts. Future prospective studies are needed to compare this approach with other HCT platforms.
MeSH term(s)	Humans ; Young Adult ; Adult ; Fanconi Anemia/therapy ; Fanconi Anemia/complications ; Retrospective Studies ; Transplantation, Homologous/adverse effects ; Transplantation Conditioning/methods ; T-Lymphocytes ; Hematopoietic Stem Cell Transplantation/methods ; Leukemia, Myeloid, Acute ; Graft vs Host Disease/etiology
Language	English
Publishing date	2023-09-29
Publishing country	England
Document type	Journal Article ; Research Support, N.I.H., Extramural
ZDB-ID	632854-4
ISSN	1476-5365 ; 0268-3369 ; 0951-3078
ISSN (online)	1476-5365
ISSN	0268-3369 ; 0951-3078
DOI	10.1038/s41409-023-02113-1
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Article ; Online: Daratumumab for the management of autoimmune cytopenias in children and young adults: a case series.

Khandelwal, Pooja / Teusink-Cross, Ashley / Kumar, Ashish R / Bleesing, Jacob J / Mehta, Parinda A / Jordan, Michael B / Chandra, Sharat / Davies, Stella M / Marsh, Rebecca A

British journal of haematology

2021 Volume 194, Issue 5, Page(s) e84–e89

MeSH term(s)	Adolescent ; Adult ; Antibodies, Monoclonal/adverse effects ; Antibodies, Monoclonal/therapeutic use ; Autoimmune Diseases/drug therapy ; Child ; Child, Preschool ; Disease Management ; Humans ; Infant ; Neutropenia/drug therapy ; Retrospective Studies ; Thrombocytopenia/drug therapy ; Young Adult
Chemical Substances	Antibodies, Monoclonal ; daratumumab (4Z63YK6E0E)
Language	English
Publishing date	2021-05-27
Publishing country	England
Document type	Case Reports ; Letter
ZDB-ID	80077-6
ISSN	1365-2141 ; 0007-1048
ISSN (online)	1365-2141
ISSN	0007-1048
DOI	10.1111/bjh.17565
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