Kooperativer Bibliotheksverbund

In: Pediatric and Developmental Pathology, SAGE Publications, Vol. 18, No. 4 ( 2015-07), p. 318-323

Abstract: Papillary thyroid carcinoma (PTC) is rare in children, although it is a known secondary malignancy after treatment for neuroblastoma (NB). The interval between NB treatment completion and PTC is usually more than 5 years. A 4-year-old, female patient with a high risk adrenal NB was found to have a 2.9-cm, right thyroid nodule on surveillance chest computed tomography (CT) 6 months after completion of her NB treatment (induction chemotherapy, tumor resection, autologous stem cell transplantation, external beam radiation to the abdominal tumor site, immunotherapy, and retinoic acid). Posttreatment surveillance included iodine-123-metaiodobenzylguanidine scans and CT scans. Fine-needle aspiration of the thyroid nodule diagnosed a follicular neoplasm, which was negative for BRAF, NRAS, KRAS, HRAS, PAX8/PPARg, and RET/PTC mutations, without evidence of metastatic NB. Nodule histology demonstrated an encapsulated follicular variant of PTC (FVPTC). Next-generation sequence analysis for a 46 cancer-gene profile was performed on both tumors with subsequent peripheral blood DNA testing. A heterozygous missense mutation in STK11 (F354L) was identified in both the NB and FVPTC. This mutation was also detected in peripheral blood mononuclear cells. Two additional heterozygous somatic missense mutations of uncertain significance were identified: KDR/VEGF receptor 2 (Q472H) on chromosome 4 and MET (N375S) on chromosome 7. To our knowledge, this is the shortest reported duration from completion of NB treatment to detection of thyroid cancer. The association of the STK11 gene with Peutz-Jeghers syndrome, lung adenocarcinomas, and medullary thyroid cancer leads to a possible association between this genetic variant and our patient's tumors.

Type of Medium: Online Resource

ISSN: 1093-5266 , 1615-5742

URL: Article

DOI: 10.2350/15-01-1597-CR.1

Language: English

Publisher: SAGE Publications

Publication Date: 2015

detail.hit.zdb_id: 1480654-X

In: JMIR Research Protocols, JMIR Publications Inc., Vol. 10, No. 5 ( 2021-5-21), p. e27650-

Abstract: Sickle cell anemia (SCA) is a genetic blood disorder that puts children at a risk of serious medical complications, early morbidity and mortality, and high health care utilization. Until recently, hydroxyurea was the only disease-modifying treatment for this life-threatening disease and has remained the only option for children younger than 5 years. Evidence-based guidelines recommend using a shared decision-making (SDM) approach for offering hydroxyurea to children with SCA (HbSS or HbS/β0 thalassemia) aged as early as 9 months. However, the uptake remains suboptimal, likely because caregivers lack information about hydroxyurea and have concerns about its safety and potential long-term side effects. Moreover, clinicians do not routinely receive training or tools, especially those that provide medical evidence and consider caregivers’ preferences and values, to facilitate a shared discussion with caregivers. Objective The aim of this study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that considers medical evidence and parent values and preferences). Methods We designed our study to compare the effectiveness of two methods for disseminating hydroxyurea guidelines to facilitate SDM: a clinician pocket guide (ie, usual care) and a clinician hydroxyurea SDM toolkit (H-SDM toolkit). Our primary outcomes are caregiver reports of decisional uncertainty and knowledge of hydroxyurea. The study also assesses the number of children (aged 0-5 years) who were offered and prescribed hydroxyurea and the resultant health outcomes. Results The Ethics Committee of the Cincinnati Children’s Hospital Medical Center approved this study in November 2017. As of February 2021, we have enrolled 120 caregiver participants. Conclusions The long-term objective of this study is to improve the quality of care for children with SCA. Using multicomponent dissemination methods developed in partnership with key stakeholders and designed to address barriers to high-quality care, caregivers of patients with SCA can make informed and shared decisions about their health. Trial Registration ClinicalTrials.gov NCT03442114; https://clinicaltrials.gov/ct2/show/NCT03442114 International Registered Report Identifier (IRRID) DERR1-10.2196/27650

Type of Medium: Online Resource

ISSN: 1929-0748

URL: Article

DOI: 10.2196/27650

Language: English

Publisher: JMIR Publications Inc.

Publication Date: 2021

detail.hit.zdb_id: 2719222-2

In: Blood, American Society of Hematology, Vol. 140, No. Supplement 1 ( 2022-11-15), p. 7875-7876

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2022-167241

Language: English

Publisher: American Society of Hematology

Publication Date: 2022

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: American Psychologist, American Psychological Association (APA), Vol. 78, No. 2 ( 2023-02), p. 107-118

Type of Medium: Online Resource

ISSN: 1935-990X , 0003-066X

URL: Article

DOI: 10.1037/amp0001086

Language: English

Publisher: American Psychological Association (APA)

Publication Date: 2023

detail.hit.zdb_id: 209464-2

detail.hit.zdb_id: 2065890-4

SSG: 5,2

In: Blood, American Society of Hematology, Vol. 134, No. Supplement_1 ( 2019-11-13), p. 4713-4713

Abstract: Background: The FDA approved hydroxyurea (HU) for the treatment of sickle cell disease (SCD) in children because it was deemed a safe and efficacious treatment. HU modifies the course of the disease, reduces complications, improves survival, and has few long-term side effects. Despite these benefits, HU uptake remains low for young children. Over 75% of patients who could benefit from HU do not receive the treatment. NHLBI clinical practice guidelines recommend use of shared decision making for HU initiation but currently, there is no "gold standard" for hematology providers to follow when beginning their discussion about HU. Thus, there is likely a gap between care guidelines and clinical practice. A first step in closing this gap is to better understand the current practice utilized by hematology providers when discussing HU as a therapeutic option. Objectives: The goal of the present study is to describe current practice used by hematology providers when discussing HU with parents of young children with SCD (0 - 5 years of age), Our primary aims are: 1) Map the process of offering HU to identify common themes, overlaps, and variations, 2) Examine the impact of a brief video presentation about the NHLBI HU guidelines on provider knowledge and comfort levels. Methods: The dissemination of methods to increase adherence to NHLBI HU guidelines are being evaluated as part of a clinical trial (NCT03442114). Hematology providers at 6 children's hospitals serving young patients with SCD completed process maps that described their current practice for discussing HU initiation with parents. Twenty five hematology providers at 10 institutions across the United States viewed a video didactic presentation on the NHLBI HU guidelines for SCD. Knowledge and comfort regarding discussing HU was assessed using a 10-point scale before and after the video. We also collected data on provider demographics, years practicing, and percentage of patients seen with SCD each week in their practice. Results: Preliminary analyses identified common themes (see Table 1 for provider characteristics). All 6 sites reported that labs and vitals were taken prior to the visit so they could be reviewed with the family. A medical doctor, nurse practitioner, or licensed practical nurse (LPN) led the HU initiation discussion. The majority of sites give their families HU-related materials to take home after the discussion and followed-up regarding the family's decision at the next clinic visit (i.e. planned for a two-visit HU initiation process). Variations identified included providing HU-related materials to the family prior to the visit, when the discussion would occur, and whether NHLBI HU guidelines framed the discussion (Figure 1). Paired samples t-tests assessed for change in medical provider reported HU knowledge and comfort before and after the SCD didactic presentation. Data revealed that there were no differences in provider comfort,t(21) = .77, p = .45, d = .03. In contrast, there was a trend towards a significant increase in medical provider knowledge from before (M = 8.4, SD = 2.3) to after (M = 8.7, SD = 2.1) the presentation, t(21) = 1.8, p = .08, d = .11, small effect. Correlation analyses determined that higher baseline provider knowledge and comfort with HU were significantly correlated with seeing more SCD patients on a weekly basis (Figure 2). Discussion: This study identified common elements of clinical practice for HU initiation in young children with SCD, but variations were also revealed. All institutions in the study obtained lab work prior to the discussion with the family; however the provider initiating the discussion varied by site (e.g., 1 site used an LPN). Most institutions plan for a two-visit discussion as families may not be ready to make a decision during the first visit but feel more supported over time. It is critical that patients and families make their decision based on SCD-specific HU information. Study data indicated that a brief didactic presentation elicited a small improvement provider HU knowledge. Moreover, exposure to the SCD population was strongly related to knowledge and comfort with HU, suggesting that hematology providers with more experience working with patients with SCD may be in the best position to initiate discussions about HU. The incorporation of decision support tools might help to support hematology providers and reduce the variation across institutions observed in our study. Disclosures King: Tioma Therapeutics (formerly Vasculox, Inc.):: Consultancy; RiverVest: Consultancy; Novimmune: Research Funding; WUGEN: Equity Ownership; Celgene: Consultancy; Cell Works: Consultancy; Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Amphivena Therapeutics: Research Funding; Bioline: Consultancy; Incyte: Consultancy. Piccone:Hemex Health, Inc.: Patents & Royalties. Neumayr:Terumo: Research Funding; Apopharma: Consultancy; PCORI: Research Funding; NHLBI: Research Funding; Bluebird Bio: Research Funding; Sancillo: Research Funding; Seattle Children's Research Grants: Research Funding; Doris Duke Foundation: Research Funding; Novartis: Research Funding; Bayer: Consultancy; Celgene: Research Funding; Imara: Research Funding; Sangamo: Research Funding; Silarus: Research Funding; Pfizer: Consultancy, Research Funding; Emmaus: Consultancy; CTD Holdings: Consultancy; GBT: Research Funding; La Jolla Pharmaceuticals: Research Funding; HRSA: Research Funding; CDC: Research Funding. Meier:CVS Caremark: Consultancy.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2019-124835

Language: English

Publisher: American Society of Hematology

Publication Date: 2019

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: American Journal of Hematology, Wiley, Vol. 92, No. 9 ( 2017-09)

Abstract: Nephropathy is a common and progressive complication of sickle cell anemia (SCA). In SCA mice, we found that hyperangiotensinemia in the absence of hypertension underlies nephropathy, and its downregulation by losartan, an angiotensin‐II‐receptor‐1 blocker, reduced albuminuria and progression of nephropathy. Therefore, we performed a phase‐2 trial of oral losartan, given for 6 months, to explore whether it reduced albuminuria in children and adults with SCA. Participants were allocated to groups defined by class of baseline urinary albumin‐to‐creatinine ratio (UACR): no albuminuria (NoA), microalbuminuria (MicroA), and macroalbuminuria (MacroA). The primary endpoint was a ≥25% reduction UACR from baseline. There were 32 evaluable participants (mean age 24 years; NoA = 14, MicroA = 12, MacroA = 6). The primary endpoint was met in 83% of the MacroA group ( P   〈  0.0001) and 58% of the MicroA group ( P   〈  0.0001). Median fold‐change in UACR was −0.74 for MacroA and −0.46 for MicroA. In MacroA and MicroA, UACR classification improved in 50% but worsened in 11%. Urine osmolality and estimated glomerular filtration rate (eGFR) did not change significantly. Losartan was discontinued in three participants [leg cramps, N  = 1; decline in eGFR 〉 25% (142➝104 mL/minute/1.73 m 2 ), N  = 1; rise in serum creatinine 〉 50% (0.2➝0.3 mg/dL), N  = 1]. Albuminuria was associated with diastolic dysfunction and impaired functional capacity, although cardiopulmonary status was unchanged after 6 months of losartan therapy. In summary, losartan decreased urinary albumin excretion in most participants with albuminuria. Those with macroalbuminuria had the greatest benefit. This study forms the basis for a phase‐3, randomized, placebo‐controlled trial of losartan for the nephropathy of SCA.

Type of Medium: Online Resource

ISSN: 0361-8609 , 1096-8652

URL: Issue

URL: Article

DOI: 10.1002/ajh.v92.9

DOI: 10.1002/ajh.24810

Language: English

Publisher: Wiley

Publication Date: 2017

detail.hit.zdb_id: 1492749-4

In: Pediatric Blood & Cancer, Wiley, Vol. 65, No. 12 ( 2018-12)

Type of Medium: Online Resource

ISSN: 1545-5009 , 1545-5017

URL: Issue

URL: Article

DOI: 10.1002/pbc.v65.12

DOI: 10.1002/pbc.27398

Language: English

Publisher: Wiley

Publication Date: 2018

detail.hit.zdb_id: 2130978-4

In: Journal of Pediatric Psychology, Oxford University Press (OUP), Vol. 44, No. 10 ( 2019-11-01), p. 1196-1204

Abstract: Adolescents with sickle cell disease (SCD) are at increased risk for complications. Hydroxyurea is a medication that can ameliorate risk but to benefit, adolescents must adhere to treatment. Study aims were to describe how adolescents and their caregivers decided who was responsible for treatment tasks, to describe adolescents’ and caregivers’ responsibility for these tasks, and to examine if hydroxyurea adherence was associated with younger adolescent age, less discrepancy between adolescents’ and caregivers’ reports of adolescent responsibility, and higher caregiver involvement. Methods Twenty-nine dyads completed treatment responsibility measures. A combination of laboratory and electronic prescription data were used to determine hydroxyurea adherence and electronic medical records were used to determine appointment adherence. Results Few dyads agreed or planned how to complete treatment tasks. Adolescents shared responsibility with caregivers for medication-taking tasks. Adolescents perceived caregivers and caregivers perceived adolescents were overall responsible for treatment, especially for appointment tasks. Half of adolescents were adherent to hydroxyurea and half were adherent to appointments but medication adherence was not associated with age, discrepancy between adolescents’ and caregivers’ responses, or caregiver involvement. Conclusions Despite frequent hydroxyurea and appointment nonadherence, few adolescents and caregivers plan how to manage adolescents’ SCD treatment or perceive they are overall responsible. Future studies are needed to determine the factors that influence these perceptions and if increasing adolescent and caregiver treatment planning improves adherence and clinical outcomes.

Type of Medium: Online Resource

ISSN: 0146-8693 , 1465-735X

URL: Article

DOI: 10.1093/jpepsy/jsz061

Language: English

Publisher: Oxford University Press (OUP)

Publication Date: 2019

detail.hit.zdb_id: 1470323-3

SSG: 5,2

In: Blood, American Society of Hematology, Vol. 136, No. Supplement 1 ( 2020-11-5), p. 26-27

Abstract: Background: Sickle cell disease (SCD) is a genetic disorder that causes significant medical and neurologic morbidity in children. Hydroxyurea (HU) is the primary medication used to prevent these complications. National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend offering HU to children as young as 9 months of age with SCD (HbSS or HbSB0 thalassemia) using a shared decision-making approach. Although HU has proven efficacious it remains underutilized and caregivers report that they are not always actively involved in the decision to initiate this therapy. Reasons for limited HU uptake likely include lack of clinician knowledge and training and negative caregiver perceptions. Thus, we developed the Engage-HU trial as a novel approach to address HU utilization barriers. A critical consideration for this trial was that SCD primarily affects individuals of African and Hispanic/Latino descent. In these minority populations, intervention trials are sometimes terminated early because of recruitment difficulties related to mistrust of research, caregiver burden, and transportation issues. As such, the Engage-HU trial design included best-practice strategies for recruiting people of color in research. This study describes these strategies, the initial recruitment plan, preliminary recruitment outcomes and strategies, and our procedural adaptations. Study Design and Methods: Engage-HU is a randomized control trial (NCT03442114) to assess how clinicians can engage caregivers in a shared discussion that considers their values and preferences and includes evidence that supports HU. Engage-HU compares two dissemination methods for clinicians to facilitate shared decision-making with caregivers of young children with SCD: 1) the American Society of Hematology Pocket Guide, and 2) the HU Shared-Decision Making (H-SDM) Toolkit. The study aims to recruit 174 caregivers and evaluate the effectiveness of the dissemination methods on patient-centered outcomes (caregiver confidence in decision-making and perceptions of experiencing shared decision-making) as well as HU uptake and child health outcomes. Eligible children are aged 0 to 5 years, candidates for HU, and their caregiver has not made a decision about HU in the past 3 months. The trial is being conducted at 9 sites in the United States and uses a stepped-wedge design. Data will be analyzed based on the intent-to-treat principle. All participants will remain in the arm of the study to which they were randomized, regardless of whether or not they receive the assigned dissemination method. The primary endpoints are caregiver decisional uncertainty and caregiver perception of shared decision-making measured using validated tools. Data will be analyzed using a linear mixed effects regression model with a robust variance estimator and maximum likelihood estimation with observations clustered within site. The Engage-HU trial includes adaptations to increase recruitment such as tailored messaging, a relational recruitment approach, streamlined data collection, and a Stakeholder Advisory Committee. However, even with these adaptations, the first 6-months of the trial yielded lower than anticipated recruitment. Rather than terminate the trial or accept low enrollment, the research team implemented a series of recruitment strategies to address barriers including helping to improve research coordinator knowledge of the study purpose and adjusting no-show and follow-up procedures (e.g., calls to families after missed appointments and reminder calls before appointments). Site clinicians and clinic staff were provided with additional training so they could give more context about Engage-HU to caregivers and the study principal investigator led monthly "all coordinator" calls to provide support by sharing updates and experiences about successful recruitment. Implementation of these strategies resulted in triple the number of enrollments over the next 7-months compared to the previous 6-months (Table 1). Our goal in sharing this information is to provide lessons learned that can be implemented in future trials with the systematically underserved SCD population. It is also anticipated that methods described here may also inform clinical approaches to better engage caregivers of young children around critical clinical conversations, such as initiating medications like HU. Disclosures King: Magenta Therapeutics: Membership on an entity's Board of Directors or advisory committees; Bioline: Consultancy; RiverVest: Consultancy; Novimmune: Research Funding; Celgene: Consultancy; Tioma Therapuetics: Consultancy; Amphivena Therapeutics: Research Funding; WUGEN: Current equity holder in private company; Cell Works: Consultancy; Incyte: Consultancy. Smith-Whitley:Prime: Other: Education material; Celgene: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees. Neumayr:Emmaus: Consultancy; Bayer: Consultancy; CTD Holdings: Consultancy; Pfizer: Consultancy; ApoPharma: Consultancy, Membership on an entity's Board of Directors or advisory committees; Micelle: Other: Site principal investigator; GBT: Other: Site principal investigator; PCORI: Other: site principal investigator; Novartis: Other: co-investigator; Bluebird Bio: Other: co-investigator; Sangamo Therapeutics: Other; Silarus: Other; Celgene: Other; La Jolla Pharmaceuticals: Other; Forma: Other; Imara: Other; National Heart, Lung, and Blood Institute: Other; Health Resources and Services Administration: Other; Centers for Disease Control and Prevention: Other; Seattle Children's Research: Other. Yates:Novartis: Research Funding. Thompson:Novartis: Consultancy, Honoraria, Research Funding; CRISPR/Vertex: Research Funding; BMS: Consultancy, Research Funding; Baxalta: Research Funding; Biomarin: Research Funding; bluebird bio, Inc.: Consultancy, Research Funding.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2020-141471

Language: English

Publisher: American Society of Hematology

Publication Date: 2020

detail.hit.zdb_id: 1468538-3

detail.hit.zdb_id: 80069-7

In: Blood, American Society of Hematology, Vol. 138, No. Supplement 1 ( 2021-11-05), p. 1891-1891

Abstract: Background: Hydroxyurea (HU) is the primary medication used to prevent the significant medical and neurologic morbidities of pediatric sickle cell disease (SCD; HbSS or HbSB0 thalassemia). Despite the benefits of HU, it remains under-utilized likely due to lack of clinician knowledge/training and negative caregiver perceptions. Thus, we developed the Engage-HU randomized controlled trial (NCT03442114) as a novel approach to address HU utilization barriers. Engage-HU is designed to assess how clinicians can engage caregivers in a shared discussion that considers their values, preferences, and scientific evidence about HU. The COVID-19 pandemic has resulted in significant changes to healthcare delivery for children with SCD, as they are at increased risk of severe illness from COVID-19 infection. Given their risk status, it was recommended that patients with SCD complete telehealth visits when possible. Some families also chose to delay care because they feared their child would get infected at hospitals/healthcare clinics that care for COVID-19 positive patients. Since the lives of all families enrolled in the Engage-HU trial have been affected to some extent, we incorporated measures to capture the impact of the COVID-19 pandemic and the usability of telemedicine implementation and services. Methods: Engage-HU is a randomized control trial comparing two dissemination methods for clinicians to facilitate shared decision-making with caregivers of young children with SCD. Study outcomes include caregiver confidence in decision-making and perceptions of experiencing shared decision-making as well as HU uptake and child health outcomes. Eligible children are 0 to 5 years, candidates for HU, and their caregiver has not decided about HU in the past 3 months. The trial is being conducted at 9 sites in the United States and uses a unidirectional crossover design. The primary endpoints are caregiver decisional uncertainty and caregiver perception of shared decision-making measured using validated tools. Data will be analyzed using the intent-to-treat principle, and all participants will remain in the arm to which they were randomized. A multiple group comparison analysis will be performed to assess significant response variable differences by group randomization. The Engage-HU study aims to recruit 174 caregivers who are considering initiating HU. The trial is being conducted at 9 sites in the United States. Data collection is ongoing, and 160 caregiver-participants have been enrolled to date. Since May 2020, caregiver-participants have completed the COVID-19 Exposure and Family Impact Scales (CEFIS), which contain 2 subscales (exposure to potentially traumatic aspects of the pandemic, impact on families), and the COVID-19 telemedicine use survey during a study visit. Results: Currently, 8 of the 9 sites have collected data from 48 caregivers (93.8% mothers), most of whom (93.8%) identify as African American/Black (see Figure 1). Correlations indicated that older caregivers experienced greater exposure (Mean = 7.0, SD = 4.1, range = 1-19) to potentially traumatic aspects of the pandemic (r = .31, p = .04). Distress related to COVID-19 varied widely across the sample, for both caregivers (Mean = 5.9, SD = 2.9, range = 1-10) and children (Mean = 4.1, SD = 3.4, range = 1-10). Scores on the telemedicine usability survey were generally high, indicating that caregivers are happy with the quality of care delivered via telehealth. However, caregivers (r = .30, p = .09) and children (r = .32, p = .07) experiencing more pandemic-related distress reported less satisfaction with telehealth. Conclusion: Although Engage-HU has resumed research operations, recruitment has not reached pre-pandemic targets, as fewer eligible patients are scheduled for routine care visits at SCD clinics. Our preliminary analyses suggest a significant continued impact of the pandemic on families and general satisfaction with the quality of healthcare delivered via telemedicine. These findings indicate that targeted screenings to identify and intervene for those who demonstrate more COVID-19 pandemic-related distress are needed. Figure 1 Figure 1. Disclosures Quinn: Forma Therapeutics: Consultancy; Aruvant: Research Funding; Novo Nordisk: Consultancy; Emmaus Medical: Research Funding. Yates: Agios Pharmaceuticals: Current Employment. Badawy: Sanofi Genzyme: Consultancy; Vertex Pharmaceuticals Inc: Consultancy; Bluebird Bio Inc: Consultancy. Thompson: bluebird bio, Inc.: Consultancy, Research Funding; Baxalta: Research Funding; Biomarin: Research Funding; Celgene/BMS: Consultancy, Research Funding; CRISPR Therapeutics: Research Funding; Vertex: Research Funding; Editas: Research Funding; Graphite Bio: Research Funding; Novartis: Research Funding; Agios: Consultancy; Beam: Consultancy; Global Blood Therapeutics: Current equity holder in publicly-traded company. Smith-Whitley: Global Blood Therapeutics: Current Employment. King: National Cancer Institute: Research Funding; National Heart, Lung, and Blood Institute: Research Funding; Health Resources and Services Administration: Research Funding; Global Blood Therapeutics: Research Funding. Meier: CVS Caremark: Consultancy; Forma Therapeutic: Membership on an entity's Board of Directors or advisory committees; NovoNordisk: Membership on an entity's Board of Directors or advisory committees; Novartis,: Other: Data Safety Monitoring Board membership; NHLBI: Other: Data Safety Monitoring Board membership; Global Blood Therapeutics: Other: Steering Committee membership, grant funding; CDC,: Other: grant funding; Indiana Department of Health: Other: grant funding . Tubman: Global Blood Therapeutics: Consultancy, Research Funding; Novartis Pharmaceuticals: Honoraria, Research Funding; Forma Pharmaceuticals: Consultancy; Perkin Elmer: Honoraria. Crosby: Forma Therapeutics: Honoraria; PCORI: Research Funding; HRSA: Research Funding; Global Blood Therapeutics Panel: Honoraria; Children's Hospital of Philadelphia: Honoraria; Professional Resource Exchange: Patents & Royalties: $30-$60 every other year; SCDAA: Honoraria; NHLBI: Other: Payment for review of LRP Proposals, Research Funding. OffLabel Disclosure: Hydroxyurea has been FDA approved for the treatment of sickle cell disease for patients ages 2 years and above but NHLBI and ASH Guidelines recommend it be offered to children as young as age 9 months.

Type of Medium: Online Resource

ISSN: 0006-4971 , 1528-0020

URL: Article

DOI: 10.1182/blood-2021-152722

Language: English

Publisher: American Society of Hematology

Publication Date: 2021

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detail.hit.zdb_id: 80069-7