Cytomegalovirus retinitis (CMVR) commonly affects immunocompromised individuals, including acquired immunodeficiency syndrome (AIDS), post-organ transplant recipients and allogeneic stem cell ...transplant recipients. CMVR occurring in the acute lymphoblastic leukemia (ALL) maintenance phase of chemotherapy is rare and has been described in the literature as isolated case reports or case series. We report a case of unilateral CMVR in a pediatric patient during maintenance phase therapy for ALL. A 14-year-old boy known case of T-cell ALL with CNS2a status, was treated according to the Children’s Oncology Group (COG) AALL0434 protocol. Induction therapy consisted of the standard high-risk four drugs, in addition to intrathecal methotrexate. At week 166 of maintenance therapy, the child presented with painless progressive loss of vision in the right eye for one week. The best-corrected visual acuity (BCVA) of the right eye was 6/36 and the left eye was 6/6. Dilated fundus examination of the right eye showed multiple large yellow-white cloudy chorioretinal lesions with areas of intraretinal hemorrhages in the macula, and overlaying focal vitritis. Optical coherence tomography (OCT) of the right eye showed macular edema and mild subretinal fluid. Cytomegalovirus polymerase chain reaction of the blood was detected with high quantitative value. A diagnosis of CMVR was made and an induction doses of intravenous ganciclovir was followed by maintenance doses of oral valganciclovir. Our case suggests that pediatric patients with ALL in the maintenance phase are considered immunocompromised and that physicians should be aware of CMVR incidence in such group. Early diagnosis and prompt treatment are important to preserve vision and prevent future visual morbidity.
Many patients with sickle cell disease (SCD) need surgical management during their lifetime. The best approach for preoperative transfusion in SCD is still to be determined. In this single-center ...retrospective study, we included HBSS/HBS-Beta
0
-thalassemia patients younger than 16 years of age who underwent surgery between January 2008 and July 2019. Preoperative transfusion assignment (PTA) was based on SCD severity and surgical risk. Patients were assigned to no transfusion, simple transfusion, or exchange transfusion. A total of 284 patients were identified and 66 (23%) underwent 78 procedures. Mean age at the time of procedure was 8 (5-11) years, mean baseline hemoglobin was 8.5 (7.8-9.3) g/dl, and mean hemoglobin F was 18.4 ± 8.2%. SCD severity was low-risk in 57 (73%) and high-risk in 21 (27%) patients. Surgical risk was low-risk in 20 (25.6%) and medium-risk in 58 (74.4%) procedures. PTA was no transfusion in 17 (22%), simple transfusion in 40 (51%), and exchange transfusion in 21 (27%) procedures. Postoperative complications occurred in five (6.4%) of procedures only in the simple transfusion group (three acute chest syndrome, one hemolytic anemia, one pain crisis) undergoing medium-risk surgery. Preoperative risk-based transfusion assignment is feasible. Despite a high baseline hemoglobin level in the no transfusion group, none of the patients developed postoperative complications. It is possible that the high baseline hemoglobin F phenotype was protective and indicates the need to study the risk/benefit of interventions used in this phenotype.
Celotno besedilo
Dostopno za:
DOBA, IJS, IZUM, KILJ, NUK, PILJ, PNG, SAZU, UILJ, UKNU, UL, UM, UPUK
e18797
Background: Patients with cancer are vulnerable population that suffered during the COVID-19 pandemic from SARS-CoV-2 infection and from the pandemic’s impact on healthcare systems. We are ...presenting the findings of MENA Registry for COVID-19 and Cancer (MRCC) regarding the SARS-CoV-2 infection presentation, diagnosis, treatment, complications, and outcomes. Methods: MRCC was adapted from ASCO COVID-19 Registry and included patients with SARS-CoV-2 infection and underlying cancer diagnosis including a newly diagnosed cancer in the work-up phase or patients with active cancer receiving cancer therapy or supportive care, or within first year of adjuvant chemotherapy or after one year of curative therapy and receiving hormonal therapy. Registry included data on patients from 12 centers in eight countries in the MENA region, namely: Saudi Arabia, Jordan, Lebanon, Turkey, Egypt, Algeria, United Arab Emirates, and Morocco. The data included patient and disease characteristics, COVID-19 presentation, management, and outcomes. The follow up is differential as data get captured at different points of disease trajectory for each patient which may not reflect the final outcome. Results: Data on 1345 patients were captured in the study by December 7, 2021. Median age was 57.1 years (18-98), whereas 56.1% were females. The median follow-up was 98.5 days (0-554). The most common COVID-19 symptoms was fever (50.3%) and 26.8% of patients were asymptomatic. Out of the 959 patients with complete data on hospitalization, 554 (57.8%) were hospitalized and 126 of them (22.7%) were admitted to intensive care unit (ICU). The majority of hospitalized patients (60%) had respiratory complications and 13.9% had sepsis and 8.5% suffered acute renal injury. As shown in Table, more than quarter of the patients died with 47% of death from COVID-19 or related complication and 60.6% died at home. More than half of the patients were fully recovered from infection. Conclusions: Although more than half of the patients recovered form COVID-19 and more are expected to recover with a longer follow up, the death toll and complications remain high in this patient population. Future analysis of the impact of vaccination and better disease management as well as the impact of newer variants would provide a useful insight on managing this vulnerable population.Table: see text
Background
Treatment modality impacts outcome of childhood low-grade glioma (LGG). Optimizing management in developing countries can be challenging. This study evaluates the clinical characteristics, ...treatment, and factors influencing outcome of childhood LGG in Saudi Arabia.
Patients and methods
This study retrospectively evaluated 59 children consecutively diagnosed with LGG between January 2001 and June 2016.
Results
Median age at diagnosis was 6.0 years. Pilocytic astrocytoma represented 64.9% of cases. The anatomic site was cerebellar in 23.7%, cerebral in 18.6%, hypothalamic-optic pathway in 33.9%, and midline in 23.7%. The 5-year overall survival (OS) and progression-free survival (PFS) were 90.6 ± 4.7 and 54.3 ± 8.4%, respectively. Initial treatment was observation in 28.8%, surgery alone in 35.6%, chemotherapy in 13.6%, radiotherapy in 5.1%, and combined in 16.9% of cases. The corresponding 5-year PFS was 56.3 ± 15.6, 53.3 ± 14.0, 22.9 ± 19.7, 33.3 ± 27.2, and 88.9 ± 10.5%, respectively (
p
= 0.006). Among the 61% who had surgical intervention (either alone or in combination with other therapies), 22% achieved complete resection with 5-year radiation/progression-free survival (RPFS) of 87.5 ± 11.7% compared to 27.6 ± 10.8% for subtotal resection/biopsy and 62.2 ± 17.0% for no surgery (
p
= 0.013). Adjuvant therapy for residual tumor improved survival with 5-year PFS of 66.7 ± 19.2% for chemotherapy and 100% for radiotherapy compared to 12.5 ± 11.4% for observation (
p
= 0.033).
Conclusions
We identified variability in the outcomes of LGG. Fewer surgeries with lower rates of total resection were noted, compared to reports from international cooperative groups. The extent of resection was predictive of RPFS. Adjuvant therapy improved the outcome of patients with residual disease, resulting in PFS rates comparable to international data.
Background: SCD is an inherited blood disorder that for many patients (pts) has a high clinical burden, results in poor quality of life (QoL), and reduces life expectancy. Gaining a deeper ...understanding of pt and HCP experiences of SCD is important to improve pt management.
Aims: SWAY was a cross-sectional survey that assessed pt and HCP experiences of SCD. Here we focus on the experiences of HCPs from various regions on SCD symptoms and complications, impact of SCD on QoL, treatment goals and treatment satisfaction.
Methods: SWAY was developed by international SCD expert physicians, pt advocates and Novartis. HCPs completed the survey between Apr and Oct 2019. Eligible HCPs had qualified in their primary specialty by 2014 and were managing ≥10 SCD pts at the time of survey (≥5 pts per HCP in Canada; ≥2 pts in the Netherlands). Responses to questions on how much SCD impacts pt QoL, and on HCP treatment satisfaction, were ranked on a Likert scale (1-7, where 1=not at all/strongly dissatisfied, 7=a great deal/strongly satisfied; 5-7 indicated high impact/satisfaction). The data reflect only the experiences of the surveyed HCPs in each region (recruited by Adelphi Real World fieldwork). A limitation is that Asia and South America (SA) were represented by single countries (India and Brazil, respectively).
Results: SWAY was completed by 365 HCPs from 6 regions (Table). In all regions HCPs recognized the prevalence of acute and chronic pain, however acute pain was reported less frequently by HCPs in Africa than in other regions (Table). Acute chest syndrome and joint issues were among the top 5 most frequently mentioned complications by HCPs in all regions. Globally, HCPs recognized the high impact of SCD symptoms and complications on pt QoL and the high negative impact of SCD on pt emotional wellbeing (Likert score 5-7 reported by 79-100% and 71-97%, respectively).
Fewer HCPs in the Middle East (ME) reported a high impact of SCD on physical and sexual activity, compared with HCPs in other regions. Around 40% of HCPs in the ME and Asia thought SCD has a high impact on daily activities, compared with 79-90% of HCPs in other regions. In Asia, fewer HCPs reported that SCD has a high impact on pts' education and ability to maintain a job compared with HCPs in other regions (Figure).
Hydroxyurea (HU) was among the top 3 most common therapies ever initiated and was the therapy most likely to be initiated in any age group by HCPs in almost all regions. In Africa, the most common therapy ever initiated and the therapy most likely to be initiated in any age group was opioids (Table). Fewer HCPs in North America (NA; 32%) and SA (27%) were highly satisfied with current SCD treatments, compared with HCPs in other regions (46-72%). The main reason for dissatisfaction was limited treatment options in all regions except Asia, where HCPs said they were unable to reach their treatment goals with current therapies.
Improving pts' QoL was among the top 3 treatment goals for 51-84% of HCPs across all regions. For HCPs in NA and the ME, the most important goal when treating vaso-occlusive crises was to improve QoL; in SA, Europe and Asia it was to avoid organ damage; and in Africa it was to eliminate pain completely.
Discussion: The top 5 most frequent SCD symptoms and complications that HCPs reported were similar across all regions. There were regional differences in HCP experiences of how SCD impacts aspects of pts' daily life, with fewer HCPs in the ME reporting a high impact on physical and sexual activity, and fewer HCPs in Asia and the ME reporting a high impact on daily activities compared with other regions. This may be due to cultural variations, with pts in these regions being less comfortable discussing these topics with HCPs. There was a difference in the reported impact of SCD on school and work between HCPs in Asia and other regions, which could be due to varying expectations regarding school/work productivity. HU was one of the top 3 most common treatments ever initiated by HCPs for pts of any age, except in Africa, which may be due to an educational knowledge gap about HU, high cost, or poor access in this region. HCPs in almost all regions, except Asia, were dissatisfied with current SCD treatments because of limited therapeutic options, indicating a global unmet need for additional treatment choices. Improving QoL was the most important treatment goal for HCPs in all regions, demonstrating the high negative impact that SCD has on pt QoL and the ongoing need for methods to address this.
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Osunkwo: Novartis Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau; FORMA Therapeutics: Consultancy; Global Blood Therapeutics: Consultancy, Speakers Bureau; Chiesi: Consultancy; Acceleron: Consultancy; Cyclerion: Consultancy; Emmaus: Consultancy. Minniti: Roche: Consultancy, Honoraria; NovoNordisk: Consultancy, Honoraria; Novartis: Consultancy, Honoraria; GBT: Consultancy, Research Funding. Nur: Roche: Speakers Bureau; Celgene: Speakers Bureau; Novartis: Research Funding, Speakers Bureau. Nero: Global Blood Therapeutics: Consultancy; Editas Medicine: Consultancy; bluebird bio: Consultancy; Novartis: Consultancy. Colombatti: Global Blood Therapeutics: Research Funding; Addmedica: Consultancy; Forma Therapeutics: Consultancy; Novartis: Consultancy; NovoNordisk: Consultancy; BlueBirdBio: Consultancy; Global Blood Therapeutics: Consultancy; BlueBirdBio: Research Funding. de Montalembert: Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Addmedica: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; bluebird bio: Membership on an entity's Board of Directors or advisory committees; Vertex: Membership on an entity's Board of Directors or advisory committees. Abboud: Jazz Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Research Support and Advisory Board, Research Funding; AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Other: Reserach support and advisory board , Research Funding; GBT: Other: Research Support, Research Funding; Vertex Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: DSMB. Arlet: Addmedica: Research Funding; Pfizer: Honoraria; Novartis company: Consultancy, Honoraria, Research Funding. Jastaniah: Novartis: Consultancy, Honoraria, Research Funding. Pita: GLOBAL ALLIANCE OF SCD: Membership on an entity's Board of Directors or advisory committees; LUA VERMELHA SCD ASSOCIATION: Membership on an entity's Board of Directors or advisory committees. Francis-Gibson: Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Sickle Cell Disease Association of America: Current Employment; ASH: Membership on an entity's Board of Directors or advisory committees; Alliance for Regenerative Medicine Foundation for Cell and Gene Medicine: Membership on an entity's Board of Directors or advisory committees; Global Alliance of SCD Organizations: Membership on an entity's Board of Directors or advisory committees. Trimnell: Novartis: Consultancy; Cyclerion: Consultancy; Global Blood Therapeutics: Consultancy. DeBonnett: Novartis Pharmaceuticals Corporation: Current Employment. Bailey: Novartis Pharmaceuticals: Other: I am an employee of Adelphi Real World, which received payment from Novartis Pharmaceuticals for this research. Rajkovic-Hooley: Novartis Pharmaceuticals: Other: I am an employee of Adelphi Real World, which received payment from Novartis Pharmaceuticals for this research. James: GBT: Honoraria; Novartis: Honoraria.
Standardizing Body Surface Area (BSA) determination is essential for avoiding variation in chemotherapy dosage calculations. In this study we compared variation in BSA calculation using weight and ...height by the Mosteller formula with weight alone using recently adapted table at the Princess Norah Oncology Center (PNOC).
Methods: Cross-sectional study of pediatric oncology patients presenting to the pediatric oncology clinic at PNOC over a week period of time.
Results: One hundred consecutive pediatric oncology patients presented to the clinic. The mean BSA calculated by the Mosteller formula was 0.83m2 (Standard Deviation = 0.24) and the mean BSA determined by the table (based on weight alone) was 0.82m2 (Standard Deviation = 0.25). The mean variation in dosing between the two methods was 1.64% (Standard Deviation = 3.4). Only 13 out of 100 patients (13%) had equal dosing using both methods and 21 out of 100 patients (21%) had dosing variation greater than 5%. When comparing both methods, using paired t-test, the difference was statistically significant (t(99) = 3.99 and P < 0.001).
Conclusion: Significant differences in BSA-based chemotherapy dosing exist in our center. The Mosteller method should remain the standard until prospective studies are performed to determine the significance of this dosing variability on toxicity and survival outcome.
Objectives: Acute lymphoblastic leukemia (ALL) with CNS2 status predicts inferior outcome and a high rate of CNS relapse, similar to overt CNS leukemia (CNS3). The purpose of this study was to ...determine if intrathecal (IT) dose intensification during induction would improve outcomes and reduce CNS relapse for CNS2 disease.
Methods: From January 2001 to December 2014, children (1-14 years) with newly diagnosed ALL were treated at the Princess Noorah Oncology Centre (PNOC) following modifications of the Children's Oncology Group (COG) protocols. We intensified IT methotrexate (ITM) during induction for patients with CNS2 disease. Patients were evaluated for overall survival (OS), disease-free survival (DFS), and cumulative incidence of relapse (CIR).
Results: 449 children with T-cell (14.3%) or B-cell (85.7%) ALL were treated using PNOC-SR or PNOC-HR regimens (Jan 2001- Dec 2007) or CALL08 regimens (Arm A SR, Arm B IR, and Arm C HR) (Jan 2008 - Dec 2014). The 5-year OS, DFS, and CIR were 87.2 ± 1.6%, 81.7 ± 1.9%, and 13.0 ± 1.7%, respectively. The OS and DFS of patients with CNS2 were significantly superior to that of patients with CNS3 (P = 0.025 and P = 0.019, respectively). Patients with CNS2 had similar OS and DFS to those with CNS1. None of the patients with CNS2 at initial diagnosis experienced CNS relapse.
Conclusions: ITM intensification during induction was associated with elimination of CNS recurrence in patients with CNS2 disease and childhood ALL. Controlled studies are needed to confirm this observation.
Celotno besedilo
Dostopno za:
DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, UILJ, UKNU, UL, UM, UPUK
Background: Sickle cell disease (SCD) is associated with many clinical complications, with vaso-occlusive crises (VOCs) being a hallmark of the disease. SCD-related complications are largely driven ...by vaso-occlusion and hemolytic anemia, and can lead to end-organ damage and early death. Analyses of SWAY, a cross-sectional survey, highlighted a substantial global impact of SCD on patients' quality of life (QoL) (James et al. ASH 2019; Osunkwo et al. ASH 2019). However, understanding how the burden of disease differs for pts of different ages could help improve management of SCD over a pt's lifespan.
Aim: To assess, using data from SWAY, whether symptoms (excluding VOCs, as previously analyzed by Osunkwo et al. EHA 2020), treatment goals and the perceived impact of SCD were different for pts of different ages.
Methods: Between April and October 2019, 2145 SCD pts aged ≥6 years participated in SWAY. The survey was completed by proxy (parent/caregiver/guardian) for pts aged 6-11 years and could be optionally self-completed by pts aged ≥12 years. Opinions were captured using a 1-7 Likert scale for some questions (5-7 indicated high satisfaction/impact/agreement). SWAY was not designed to assess treatment outcomes; all analyses are descriptive. Age groups were not matched and pts were not followed over time.
Results: To understand how the most dominant symptoms of SCD differ for pts of different ages, the top 5 most commonly reported symptoms, stratified by age, were analyzed (Figure 1). Fatigue and bone aches were consistently reported, and the proportion of pts reporting them trended towards increasing with age. Furthermore, when asked which symptoms they most wanted to be resolved, fatigue was ranked in the top 3 by 40.7% of pts. Anxiety was a dominant symptom for pts aged 19-25, 36-45 and 46-50 years, whereas low mood was a dominant symptom for pts aged 19-50 and ≥60 years. Poor appetite was a dominant symptom for pts aged 6-16 years. Breathing issues were a dominant symptom for pts aged 6-18, 46-50 and ≥60 years, whereas vision issues were a dominant symptom for pts aged 51-59 years only. Insomnia was dominant for pts aged 46-50.
The treatment goals that pts ranked as the most important were similar across age groups. Pts consistently included improving QoL, preventing worsening of SCD, reducing the number of VOCs and improving overall symptoms in their top 3, although the proportions of pts reporting these varied across age groups (Figure 2). Unsurprisingly, 23.0% of pts aged 12-16 years ranked increasing the ability to attend school in their top 3 goals. For pts aged 46-50 and 51-59 years, 27.5% and 20.7%, respectively, ranked reducing fatigue in their top 3 treatment goals.
The highest proportions of pts reporting a high impact of SCD on their emotional wellbeing were aged 46-50 years (74.5%) and ≥60 years (73.9%), compared with 59.6% of all pts. Similarly, the highest proportions of pts reporting a high impact of SCD on daily activities were aged 46-50 years (51.0%) and ≥60 years (60.9%), compared with 38.1% of all pts.
Limitations: These findings are based on pt and proxy reports, with potential parental bias being introduced for pediatric patients. There were variations in sample sizes, which was most noticeable for patients aged ≥46 years.
Discussion: Fatigue and bone aches were consistently reported as dominant symptoms for all ages. Other dominant symptoms that were not consistent across age groups were anxiety, low mood, poor appetite, breathing issues and vision issues. In addition to the 36-45 years and 46-50 years groups, anxiety was a dominant symptom for pts aged 19-25 years. Along with other changes that could occur at this age, a recent transition from pediatric to adult care may contribute to anxiety being a dominant symptom; however, any direct relationship between anxiety and transitioning between care systems requires further investigation. The consistent reporting of low mood among adults, but not pediatrics, may reflect the increasing burden of disease that occurs with age. This is supported by higher proportions of pts aged ≥46 years versus <45 years reporting a high impact on emotional wellbeing and daily life.
Improving QoL was consistently ranked the most important treatment goal for pts. This emphasizes, from the pt's perspective, the need for further improvements in the management of SCD.
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Colombatti:Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees; Addmedica: Membership on an entity's Board of Directors or advisory committees. James:Sickle Cell Society: Current Employment; Novartis: Honoraria. Andemariam:Hemanext: Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; CHNCT: Consultancy; CRISPR/Vertex: Consultancy, Membership on an entity's Board of Directors or advisory committees; Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees; Cyclerion: Consultancy, Membership on an entity's Board of Directors or advisory committees; Accordant: Membership on an entity's Board of Directors or advisory committees; Guidepoint: Honoraria; Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; NovoNordisk: Consultancy, Membership on an entity's Board of Directors or advisory committees; Roche: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Terumo BCT: Consultancy, Membership on an entity's Board of Directors or advisory committees; Sanofi Genzyme: Consultancy, Membership on an entity's Board of Directors or advisory committees; Imara: Research Funding; Vertex: Honoraria; Emmaus: Membership on an entity's Board of Directors or advisory committees; bluebird bio: Consultancy, Membership on an entity's Board of Directors or advisory committees. Inusa:Vertex: Research Funding; Bluebird bio: Research Funding; AstraZeneca: Honoraria, Other: Steering committee participation, Research Funding, Speakers Bureau; Global Blood Therapeutics: Honoraria, Other: Steering committee participation, Research Funding, Speakers Bureau; Novartis: Honoraria, Other: Steering committee participation, Research Funding, Speakers Bureau. El Rassi:Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Cyclerion: Research Funding; Pfizer: Research Funding; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees; Bluebird bio: Membership on an entity's Board of Directors or advisory committees. Francis-Gibson:Sickle Cell Disease Association of America: Current Employment. Nero:Bluebird bio: Consultancy; Novartis: Consultancy. Minniti:Global Blood Therapeutics: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Roche: Consultancy, Research Funding; TauTona: Consultancy, Research Funding; Bluebird bio: Consultancy, Research Funding; Emmaus: Consultancy, Research Funding; CLS Bering: Consultancy. Trimnell:Novartis: Consultancy; Cyclerion: Consultancy; Global Blood Therapeutics: Consultancy. Abboud:Amgen: Other: Travel support; Novo Nordisk: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Crispr Therapeutics: Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Eli Lilly: Research Funding; Modus Pharmaceuticals: Research Funding. Arlet:Novartis: Consultancy, Honoraria. de Montalembert:Addmedica: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bluebird bio: Honoraria, Membership on an entity's Board of Directors or advisory committees; Vertex: Honoraria, Membership on an entity's Board of Directors or advisory committees. Jain:Thalassemia and Sickle Cell Society: Other: Chief Medical Research Officer and Secretary. Jastaniah:Novartis: Consultancy, Honoraria. Nur:Novartis: Consultancy. Ramscar:Novartis Pharma AG: Current Employment. Bailey:Novartis: Other: Employee of Adelphi Real World, which has received consultancy fees from Novartis; Adelphi Real World: Current Employment. Rajkovic-Hooley:Adelphi Real World: Current Employment; Novartis: Other: Employee of Adelphi Real World, which has received consultancy fees from Novartis. Osunkwo:Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees; Terumo: Consultancy; Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; Acceleron: Membership on an entity's Board of Directors or advisory committees; FORMA Therapeutics: Membership on an entity's Board of Directors or advisory committees; Health Resources and Services Administration (HRSA): Research Funding; Patient Centered Outcomes Research Institute (PCORI): Research Funding; Data and Safety Monitoring Board (DSMB) membership for Micella Biopharma: Membership on an entity's Board of Directors or advisory committees.