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FDA approves new treatment for sickle cell disease  – First approval for this rare blood disorder in nearly 20 years

The U.S. Food and Drug Administration today approved Endari (L-glutamine oral powder) for patients age five years and older with sickle cell disease to reduce severe complications associated with the blood disorder.

“Endari is the first treatment approved for patients with sickle cell disease in almost 20 years,” said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “Until now, only one other drug was approved for patients living with this serious, debilitating condition.”

The safety and efficacy of Endari were studied in a randomized trial of patients ages five to 58 years old with sickle cell disease who had two or more painful crises within the 12 months prior to enrollment in the trial. Patients were assigned randomly to treatment with Endari or placebo, and the effect of treatment was evaluated over 48 weeks. Patients who were treated with Endari experienced fewer hospital visits for pain treated with a parenterally administered narcotic or ketorolac (sickle cell crises), on average, compared to patients who received a placebo (median 3 vs. median 4), fewer hospitalizations for sickle cell pain (median 2 vs. median 3), and fewer days in the hospital (median 6.5 days vs. median 11 days).  Patients who received Endari also had fewer occurrences of acute chest syndrome (a life-threatening complication of sickle cell disease) compared with patients who received a placebo (8.6 percent vs. 23.1 percent).

Common side effects of Endari include constipation, nausea, headache, abdominal pain, cough, pain in the extremities, back pain and chest pain.

Endari received Orphan Drug designation for this use, which provides incentives to assist and encourage the development of drugs for rare diseases.  In addition, development of this drug was in part supported by the FDA Orphan Products Grants Program, which provides grants for clinical studies on safety and/or effectiveness of products for use in rare diseases or conditions.

The FDA granted the approval of Endari to Emmaus Medical Inc.


Sickle cell sufferer receives life-transforming treatment – UK

Last December, we first met Adawale Lawal, who was offered a new form of stem cell transplant with the potential to cure him of the disease. NHS England, however, wouldn’t fund it. But he managed to raise the cash, and Adawale became the first adult in the UK to undergo the procedure.


Prodigy’s death shines light on slow progress against sickle cell disease

The death of the rap artist Prodigy (Albert Johnson, half of the duo Mobb Deep) at only 42 this week, after a lifetime of suffering from sickle cell disease, was a reminder of the devastating cost of the sometimes fatal genetic disorder — and of the failure to cure it.

It has been 61 years since the discovery of the mutation responsible for sickle cell, which affects about 100,000 people in the U.S., and 30 years since scientists found a compensatory mutation — one that keeps people from developing sickle cell despite inheriting the mutant genes.

Last year, when STAT examined the lack of progress, scientists and hospital officials were frank about one reason for it: Other genetic disorders, notably cystic fibrosis, attracted piles of money that led to cures, but sickle cell strikes the “wrong” kind of people, including African-Americans, and so has historically been starved for funds.


New Book

 The book Genetics and Global Public Health: Sickle Cell and Thalassaemia edited by Simon Dyson and Karl Atkin is now available in paperback for the first time:


Articles in the Medical Literature


J Neurosurg Pediatr. 2017 Jul 7:1-7. doi: 10.3171/2017.1.PEDS16576. [Epub ahead of print]

Effectiveness of surgical revascularization for stroke prevention in pediatric patients with sickle cell disease and moyamoya syndrome.

Yang W1, Xu R1, Porras JL1, Takemoto CM2, Khalid S1, Garzon-Muvdi T1, Caplan JM1, Colby GP1, Coon AL1, Tamargo RJ1, Huang J1, Ahn ES1.


OBJECTIVE Sickle cell disease (SCD) in combination with moyamoya syndrome (MMS) represents a rare complication of SCD, with potentially devastating neurological outcomes. The effectiveness of surgical revascularization in this patient population is currently unclear. The authors’ aim was to determine the effectiveness of surgical intervention in their series of SCD-MMS patients by comparing stroke recurrence in those undergoing revascularization and those undergoing conservative transfusion therapy. METHODS The authors performed a retrospective chart review of patients with MMS who were seen at the Johns Hopkins Medical Institution between 1990 and 2013. Pediatric patients (age < 18 years) with confirmed diagnoses of SCD and MMS were included. Intracranial stroke occurrence during the follow-up period was compared between surgically and conservatively managed patients. RESULTS A total of 15 pediatric SCD-MMS patients (28 affected hemispheres) were included in this study, and all were African American. Seven patients (12 hemispheres) were treated with indirect surgical revascularization. The average age at MMS diagnosis was 9.0 ± 4.0 years, and 9 patients (60.0%) were female. Fourteen patients (93.3%) had strokes before diagnosis of MMS, with an average age at first stroke of 6.6 ± 3.9 years. During an average follow-up period of 11.6 years, 4 patients in the conservative treatment group experienced strokes in 5 hemispheres, whereas no patient undergoing the revascularization procedure had any strokes at follow-up (p = 0.029). Three patients experienced immediate postoperative transient ischemic attacks, but all recovered without subsequent strokes. CONCLUSIONS Indirect revascularization is suggested as a safe and effective alternative to the best medical therapy alone in patients with SCD-MMS. High-risk patients managed on a regimen of chronic transfusion should be considered for indirect revascularization to maximize the effect of stroke prevention.

PMID: 28686127

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Crit Rev Food Sci Nutr. 2017 Jul 7:0. doi: 10.1080/10408398.2017.1319794. [Epub ahead of print]

The role of nutrition in the pathophysiology and management of sickle cell disease among Children: A Review of literature.

Ohemeng A1, Boadu I1.


Sickle cell disease (SCD) is one of the common inherited blood disorders in humans and has been associated with decreased dietary intake which results in poor nutritional status and impaired growth. Nutrition is one of the most important but often forgotten aspect of care of patients with chronic disorders and there have been emerging concern in literature on increased nutritional needs of SCD patients. This paper sought to review the available literature on the roles of individual nutrients in the pathophysiology and management of SCD among children. Children with SCD have been shown to exhibit sub-optimal status with respect to both macro-and micro-nutrients. Thus, nutrition could play an important role in the management of SCD. However, there is paucity of evidence coming from trials with large sample sizes to support the suggestion that supplementation with various nutrients that have been considered in this review will be helpful.

PMID: 28686043

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Health Qual Life Outcomes. 2017 Jul 5;15(1):136. doi: 10.1186/s12955-017-0713-x.

Adherence to hydroxyurea, health-related quality of life domains, and patients’ perceptions of sickle cell disease and hydroxyurea: a cross-sectional study in adolescents and young adults.

Badawy SM1,2, Thompson AA3, Lai JS4, Penedo FJ4, Rychlik K5, Liem RI3.



Sickle cell disease (SCD) patients have impaired domains of health-related quality of life (HRQOL). Hydroxyurea is safe and efficacious in SCD; however, adherence is suboptimal, and patients’ perceptions are poorly understood amongst adolescents and young adults (AYA). Study objectives were to: (1) examine patients’ perceptions of SCD and hydroxyurea; and (2) explore the relationship of their perceptions to clinical characteristics, HRQOL domains and hydroxyurea adherence.


Thirty-four SCD patients on hydroxyurea (≥6 months) participated in a single-institution study. Study measures included Brief-Illness Perceptions Questionnaire, ©Modified Morisky Adherence Scale 8-items, and Patient Reported Outcomes Measurement Information System (PROMIS®). We assessed the relationship of patients’ perceptions to hydroxyurea adherence using Wilcoxon rank-sum test, the number of hospitalizations using Kruskal-Wallis test, and the number of ED visits, adherence level, HRQOL domain scores using Spearman’s rho correlations. We conducted a sub-analysis in HbSS patients to evaluate the relationship of patients’ perceptions to laboratory markers of hydroxyurea adherence.


Participants were 59% male and 91% Black, and had a median age of 13.5 (range 12-18) years. Participants with ≥4 hospitalizations over 1-year prior (using electronic medical chart review) reported more negative perceptions of SCD-related symptoms and emotional response, and perceived hydroxyurea as less beneficial; all p-values ≤0.01. Most participants (74%) reported low hydroxyurea adherence. Participants with higher hydroxyurea adherence perceived more hydroxyurea benefits (r s = 0.44, p < 0.01) and had better emotional response to SCD (r s = -0.44, p = 0.01). In a sub-analysis of HbSS patients, perceived benefits of hydroxyurea positively correlated with HbF (r s = 0.37, p = 0.05) and MCV values (r s = 0.35, p = 0.05). Participants with more negative perceptions of SCD-related consequences, concerns, and emotional response, and fewer perceived hydroxyurea benefits reported worse fatigue (r s = 0.68; r s = 0.44; r s = 0.74; r s = -0.60), pain (r s = 0.56; r s = 0.54; r s = 0.63; r s = -0.39), anxiety (r s = 0.55; r s = 0.58; r s = 0.56; r s = -0.47), and depression (r s = 0.64; r s = 0.49; r s = 0.70; r s = -0.62), respectively, all p-values <0.05.


Dynamics influencing hydroxyurea adherence are multifactorial, and understanding patients’ perceptions is critical to overcoming adherence barriers. Patients’ favorable perceptions correlated with greater adherence and better HRQOL domain scores. Prospective evaluation of patients’ perceptions of SCD and hydroxyurea in relation adherence, HRQOL domains and clinical outcomes is warranted.

PMID: 28679417

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Pediatr Blood Cancer. 2017 Jul 4. doi: 10.1002/pbc.26713. [Epub ahead of print]

Invasive pneumococcal disease in children with sickle cell disease in the pneumococcal conjugate vaccine era.

Martin OO1, Moquist KL2, Hennessy JM2, Nelson SC2.


Invasive pneumococcal disease (IPD) in children with sickle cell disease (SCD) can be devastating. We sought to assess the impact of IPD in children with SCD since licensure of the pneumococcal conjugate vaccines (PCVs). We found 11 cases of IPD giving an incidence of 417 per 100,000 person-years, much higher than that reported in children without SCD. Although all isolates were sensitive to penicillin, 89% of isolates were nonvaccine serotypes. Further study is needed to characterize the incidence of and risk factors for the development of IPD in SCD in the PCV era to help drive better prevention strategies.

© 2017 Wiley Periodicals, Inc.

PMID: 28675587

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Cochrane Database Syst Rev. 2017 Jul 3;7:CD012380. doi: 10.1002/14651858.CD012380.pub2. [Epub ahead of print]

Interventions for chronic kidney disease in people with sickle cell disease.

Roy NB1, Fortin PM, Bull KR, Doree C, Trivella M, Hopewell S, Estcourt LJ.



Sickle cell disease (SCD) is one of the commonest severe monogenic disorders in the world, due to the inheritance of two abnormal haemoglobin (beta-globin) genes. SCD can cause severe pain, significant end-organ damage, pulmonary complications, and premature death. Kidney disease is a frequent and potentially severe complication in people with SCD.Chronic kidney disease is defined as abnormalities of kidney structure or function, present for more than three months. Sickle cell nephropathy refers to the spectrum of kidney complications in SCD.Glomerular damage is a cause of microalbuminuria and can develop at an early age in children with SCD, and increases in prevalence in adulthood. In people with sickle cell nephropathy, outcomes are poor as a result of the progression to proteinuria and chronic kidney insufficiency. Up to 12% of people who develop sickle cell nephropathy will develop end-stage renal disease.


To assess the effectiveness of any intervention in preventing or reducing kidney complications or chronic kidney disease in people with SCD (including red blood cell transfusions, hydroxyurea and angiotensin-converting enzyme inhibitor (ACEI)), either alone or in combination with each other.


We searched for relevant trials in the Cochrane Library, MEDLINE (from 1946), Embase (from 1974), the Transfusion Evidence Library (from 1980), and ongoing trial databases; all searches current to 05 April 2016. We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register: 13 April 2017.


Randomised controlled trials comparing interventions to prevent or reduce kidney complications or chronic kidney disease in people with SCD. There were no restrictions by outcomes examined, language or publication status.


Two authors independently assessed trial eligibility, extracted data and assessed the risk of bias.


We included two trials with 215 participants. One trial was published in 2011 and included 193 children aged 9 months to 18 months, and compared treatment with hydroxyurea to placebo. The second trial was published in 1998 and included 22 adults with normal blood pressure and microalbuminuria and compared ACEI to placebo.We rated the quality of evidence as low to very low across different outcomes according to GRADE methodology. This was due to trials having: a high or unclear risk of bias including attrition and detection bias; indirectness (the available evidence was for children aged 9 months to 18 months in one trial and a small and select adult sample size in a second trial); and imprecise outcome effect estimates of significant benefit or harm. Hydroxyurea versus placebo We are very uncertain if hydroxyurea reduces or prevents progression of kidney disease (assessed by change in glomerular filtration rate), or reduces hyperfiltration in children aged 9 to 18 months, mean difference (MD) 0.58 (95% confidence interval (CI) -14.60 to 15.76 (mL/min per 1.73 m²)) (one study; 142 participants; very low-quality evidence).In children aged 9 to 18 months, hydroxyurea may improve the ability to concentrate urine, MD 42.23 (95% CI 12.14 to 72.32 (mOsm/kg)) (one study; 178 participants; low-quality evidence).Hydroxyurea may make little or no difference to SCD-related serious adverse events including: incidence of acute chest syndrome, risk ratio (RR) 0.39 (99% CI 0.13 to 1.16); painful crisis, RR 0.68 (99% CI 0.45 to 1.02); and hospitalisations, RR 0.83 (99% CI 0.68 to 1.01) (one study, 193 participants; low-quality evidence).No deaths occurred in the trial. Quality of life was not reported. ACEI versus placeboWe are very uncertain if ACEI reduces proteinuria in adults with SCD who have normal blood pressure and microalbuminuria, MD -49.00 (95% CI -124.10 to 26.10 (mg per day)) (one study; 22 participants; very low-quality evidence). We are very uncertain if ACEI reduce or prevent kidney disease as measured by creatinine clearance. The authors state that creatinine clearance remained constant over six months in both groups, but no comparative data were provided (very low-quality evidence).All-cause mortality, serious adverse events and quality of life were not reported.


In young children aged 9 months to 18 months, we are very uncertain if hydroxyurea improves glomerular filtration rate or reduces hyperfiltration, but it may improve young children’s ability to concentrate urine and may make little or no difference on the incidence of acute chest syndrome, painful crises and hospitalisations.We are very uncertain if giving ACEI to adults with normal blood pressure and microalbuminuria has any effect on preventing or reducing kidney complications.This review identified no trials that looked at red cell transfusions nor any combinations of interventions to prevent or reduce kidney complications.Due to lack of evidence this review cannot comment on the management of either children aged over 18 months or adults with any known genotype of SCD.We have identified a lack of adequately-designed and powered studies, and no ongoing trials which address this critical question. Trials of hydroxyurea, ACEI or red blood cell transfusion in older children and adults are urgently needed to determine any effect on prevention or reduction kidney complications in people with SCD.

PMID: 28672087

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J Thromb Haemost. 2017 Jul;15(7):1307-1316. doi: 10.1111/jth.13717.

Prothrombotic aspects of sickle cell disease.

Sparkenbaugh E1, Pawlinski R1.


Sickle cell disease (SCD) is a hematologic disorder caused by a well-characterized point mutation in the β-globin gene. Abnormal polymerization of hemoglobin tetramers results in the formation of sickle red blood cells that leads to vascular occlusions, hemolytic anemia, vascular inflammation and cumulative, multiple organ damage. Ongoing activation of coagulation is another hallmark of SCD. Recent studies strongly suggested that hypercoagulation in SCD is not just a secondary event but contributes directly to the disease pathophysiology. In this article we summarize mechanisms leading to the activation of coagulation, review data indicating direct contribution of coagulation to the pathology of SCD and, we discuss the anticoagulation as a possible treatment strategy to attenuate the disease progression.

© 2017 International Society on Thrombosis and Haemostasis.

PMID: 28671346

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Am J Hematol. 2017 Jul 3. doi: 10.1002/ajh.24840. [Epub ahead of print]

Estimation of Glomerular Filtration Rate Using Serum Cystatin C and Creatinine in Adults with Sickle Cell Anemia.

Yee MEM1, Lane PA1, Archer DR1, Joiner CH1, Eckman JR2, Guasch A3.

PMID: 28670697

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Transfus Clin Biol. 2017 Jun 29. pii: S1246-7820(17)30078-2. doi: 10.1016/j.tracli.2017.05.016. [Epub ahead of print]

Management of delayed hemolytic transfusion reaction in sickle cell disease: Prevention, diagnosis, treatment.

Pirenne F1, Bartolucci P2, Habibi A2.


Transfusion remains a key treatment of sickle cell disease complications. However, delayed hemolytic transfusion reaction, the most serious complication of transfusion, may be life-threatening if hyperhemolysis develops. This syndrome is generally underdiagnosed because its biological and clinical features resemble those of vaso-occlusive crisis, and red blood cell antibodies are frequently absent. Further transfusions may aggravate the symptoms, leading to severe multiple organ failure and death. It is therefore essential to prevent, diagnose and treat this syndrome efficiently. Prevention is based principally on the attenuation of allo-immunization through the provision of extended-matched RBCs or the use of rituximab. However, such treatment may be insufficient. Early diagnosis might make it possible to implement specific treatments in some cases, thereby avoiding the need for secondary transfusion. Diagnosis is dependent on the knowledge of the medical staff. Finally, many treatments, including steroids, immunoglobulins, erythropoietin and eculizumab, have been used to improve outcome. Improvements in our knowledge of the specific features of DHTR in SCD should facilitate management of this syndrome.

Copyright © 2017 Elsevier Masson SAS. All rights reserved.

PMID: 28669521

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Transfusion. 2017 Jun 26. doi: 10.1111/trf.14212. [Epub ahead of print]

Medical and economic implications of strategies to prevent alloimmunization in sickle cell disease.

Gehrie EA1, Ness PM1, Bloch EM1, Kacker S1, Tobian AAR1.



The pathogenesis of alloimmunization is not well understood, and initiatives that aim to reduce the incidence of alloimmunization are generally expensive and either ineffective or unproven. In this review, we summarize the current medical literature regarding alloimmunization in the sickle cell disease (SCD) population, with a special focus on the financial implications of different approaches to prevent alloimmunization.


A review of EMBASE and MEDLINE data from January 2006 through January 2016 was conducted to identify articles relating to complications of SCD. The search was specifically designed to capture articles that evaluated the costs of various strategies to prevent alloimmunization and its sequelae.


Currently, there is no proven, inexpensive way to prevent alloimmunization among individuals with SCD. Serologic matching programs are not uniformly successful in preventing alloimmunization, particularly to Rh antigens, because of the high frequency of variant Rh alleles in the SCD population. A genotypic matching program could offer some cost savings compared to a serologic matching program, but the efficacy of gene matching for the prevention of alloimmunization is largely unproven, and large-scale implementation could be expensive.


Future reductions in the costs associated with genotype matching could make a large-scale program economically feasible. Novel techniques to identify patients at highest risk for alloimmunization could improve the cost effectiveness of antigen matching programs. A clinical trial comparing the efficacy of serologic matching to genotype matching would be informative.

© 2017 AABB.

PMID: 28653325

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Ann Clin Biochem. 2017 Jan 1:4563217713788. doi: 10.1177/0004563217713788. [Epub ahead of print]

ANNALS EXPRESS: Newborn screening for sickle cell disorders using tandem mass spectrometry (MS/MS): 3 years experience of using a protocol to detect only the disease states.

Moat S1, Rees D, George R, King L, Dodd A, Ifederu A, Ramgoolam T, Hillier S.



Tandem mass spectrometry (MS/MS) has recently become an alternative method for the newborn screening of sickle cell disorders (SCD), as it is able to detect haemoglobin (Hb) peptides following digestion of bloodspots with trypsin. Using the SpOtOn Diagnostics Reagent Kit, we previously developed a screening protocol to detect only the disease states of SCD, using action values based on the ratio between the variant Hb peptide to wild-type peptide abundances for the HbS, C, DPunjab, OArab, E and Lepore peptides.


Action values using the ratios between the wild type HbA (ßT1-3) peptides and the foetal Hb (γT2) peptide were developed to identify bloodspot samples from premature and transfused infants. An evaluation was undertaken to assess the transferability of the action values onto an additional MS/MS instrument. We report here our experience using this MS/MS protocol.


During a three year period we screened 100,456 babies and identified 10 SCD cases (1 HbS/HPFH, 5 HbSS and 4 HbS/C) and a case of HbE/ß-thalassaemia that was identified as a by-product. The Hb variant to wild-type peptide ratio action values were transferable to a second MS/MS instrument. Our protocol prevented the identification of an estimated 810 carrier infants. Gestational age-related action values for HbA to HbF peptide ratios were required to minimise the number of samples referred for second-line testing to exclude ß-thalassaemia.


MS/MS is a robust alternative screening technology for SCD, in addition it also optimises the use of equipment and expertise that currently exist in newborn screening laboratories.

PMID: 28656815

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Hematol Oncol Stem Cell Ther. 2017 Jun 15. pii: S1658-3876(17)30044-4. doi: 10.1016/j.hemonc.2017.05.008. [Epub ahead of print]

Hematopoietic stem cell transplantation for sickle cell anemia: The changing landscape.

Kassim AA1, Sharma D2.


Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative therapy for sickle cell disease (SCD); however, its use is limited by lack of suitable human leukocyte antigen (HLA)-matched donors and decreased application in older patients with significant morbidity. Myeloablative, HLA-identical sibling transplantation in children with SCD offers excellent long-term survival, with overall and event-free survival rates of 95% and 92%, respectively. However, the risk of graft-versus-host-disease, infections, infertility, and other long-term transplant complications, further limits its widespread use. Recent approaches using reduced intensity conditioning (RIC) are associated with lower toxicity, allowing extension of this modality to children and adults with significant morbidity; however, these approaches are also associated with increased risk of graft failure. The optimal RIC regimen that striking the optimal balance between maximizing the rate of stable engraftment while minimizing transplant-related morbidity and mortality is unknown. Alternative donor transplants, most prominently, partial HLA-mismatched related transplants (haploidentical), are being investigated with promising initial results. This review will discuss long-term results of HLA-matched sibling HSCT for SCD, and recent updates on HLA-matched unrelated donor and unrelated umbilical cord blood HSCT for SCD.

Copyright © 2017. Published by Elsevier B.V.

Free Article

PMID: 28641096

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J Pediatr Psychol. 2017 Jun 15. doi: 10.1093/jpepsy/jsx088. [Epub ahead of print]

Pediatric to Adult Care Transition: Perspectives of Young Adults With Sickle Cell Disease.

Porter JS1, Wesley KM1, Zhao MS1, Rupff RJ1, Hankins JS1.



The aim of this study was to explore perspectives of transition and transition readiness of young adult patients (YAs) with sickle cell disease (SCD) who have transitioned to adult health care. In all, 19 YAs with SCD (ages 18-30 years) participated in one of three focus groups and completed a brief questionnaire about transition topics. Transcripts were coded and emergent themes were examined using the social-ecological model of adolescent and young adult readiness for transition (SMART). Themes were consistent with most SMART components. Adult provider relationships and negative medical experiences emerged as salient factors. YAs ranked choosing an adult provider, seeking emergency care, understanding medications/medication adherence, knowing SCD complications, and being aware of the impact of health behaviors as the most important topics to include in transition programming. The unique perspectives of YAs can inform the development and evaluation of SCD transition programming by incorporating the identified themes.

PMID: 28637291

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Pediatr Blood Cancer. 2017 Jun 14. doi: 10.1002/pbc.26665. [Epub ahead of print]

Changes in urine albumin to creatinine ratio with the initiation of hydroxyurea therapy among children and adolescents with sickle cell disease.

Tehseen S1,2, Joiner CH1,2, Lane PA1,2, Yee ME1,2.



Renal damage is a progressive complication of sickle cell disease (SCD) that begins in childhood and may progress to renal failure and early mortality in 12% of adults with hemoglobin SS (HbSS) SCD. Early sickle nephropathy is characterized by hyperfiltration and microalbuminuria; therefore, urine albumin to creatinine ratio (ACR) is an effective screening tool for its detection.


This study investigated the effect of hydroxyurea (HU) therapy on urine ACR levels among children with SCD. A retrospective review was conducted to identify all patients with HbSS or HbSβ0 thalassemia of age 7-18 years who began HU therapy in 2011-2013; a control group of patients not on HU were matched by age and baseline hemoglobin. All urine ACR measurements ≤24 months prior to and ≥24 months after HU initiation were recorded.


There were 63 eligible patients on HU and 13 (25%) with albuminuria prior to HU initiation. Among those with baseline albuminuria, the median ACR was 96 mg/g prior to HU, 39 mg/g at 1 year (P = 0.02), and 25 mg/g at 2 years (P = 0.03). Albuminuria normalized in 37.5% (6/16) after 1 year and 61% (8/13) after 2 years of HU therapy. Among those without albuminuria prior to HU, 13% (6/47) developed albuminuria during HU therapy. Sixteen percent (13/80) of control patients had albuminuria in the beginning of study period, which normalized in 15% (two of 13) of patients at 1-year follow up.


Introduction of HU is associated with significant decreases in urine ACR in children with SCD and albuminuria.

© 2017 Wiley Periodicals, Inc.

PMID: 28612449



Sickle Cell Conferences and Events


Join Sickle Cell Partners of the Carolinas for the 4th annual conference, “Sickle Cell Disease… Let’s Talk About It”

Saturday September 9, 2017   8 am to 2 pm   Charlotte, NC

Friendship Missionary Baptist Church Conference Center 3400 Beatties Ford Road, Charlotte NC 28216

This day conference will feature a myriad of topics designed to engage patients, families and the at-large community and to build broader awareness about the challenges of sickle cell disease and how patients and families may be able to get beyond those challenges. Keynote Luncheon Speaker: Howard University President, Wayne A.I. Frederick, M.D., MBA.

Friendship Missionary Baptist Church Conference Center Charlotte, North Carolina. Cost is $5 for participants.

For more information, visit our website and register for the event at


Pediatric Sickle Cell Mini Symposium: The School-Aged Child

Saturday, September 9, 2017 7:45 a.m. to 3:15 p.m. Atlanta,GA

Who Should Attend:The conference will benefit pediatricians, family practice physicians, advanced practice providers (NP’s, PA’s), nurses, fellows, and residents. Other healthcare professionals involved in the care of pediatric patients with sickle cell disease mayfind the information useful and are welcome to attend.

The purpose of this symposium is to update pediatricians and family practitioners
on the most current research and clinical guidelines related to pediatric sickle cell
disease, particularly in the school-aged child, and to discuss key considerations
when caring for these patients.

Location & Accommodations Emory Health Sciences Research Building
1760 Haygood Drive NE Atlanta, Georgia 30322

For more information, contact


SCDAA is pleased to announce the 45th Annual National Convention on Sickle Cell Disease

With over 447 researchers, physicians, nurses, socials workers, individuals living with SCD & SCT and more we are excited to reunite with you again on October 23-28, 2017!

The SCDAA Annual Convention is a four-day conference designed to address the multi-factorial aspects of Sickle Cell Disease. This year the event will be held in Atlanta, Georgia, a city near and dear to the sickle cell community!


SCDAA Announces National Abstract Competition for the 45th Annual Convention

Sickle Cell Disease Association of America, Inc.(SCDAA) seeks to highlight the work of Researchers, Community-based Member Organizations, Physicians, Nurses, Social Workers and others working on behalf of people with sickle cell disease and their families. Individuals or organizations interested in presenting reports on work completed or in progress should submit an abstract using the link below. All approved abstracts will be published in the final program to be distributed to registered conference attendees. During peer-review, abstracts judged to be the best in their categories will be selected as national finalists.

Abstract Categories Include:

  • Community Based Research
  • Clinical Research
  • Public Health, Policy, and Psychosocial Research
  • Basic Science and Translational Research

To be eligible, abstracts must meet guidelines and be submitted by June 15, 2017 (there will be no deadline extensions). Abstracts will be reviewed and ranked by the national abstract review committee. Abstract finalists will be judged during oral presentation at convention and the “Best Abstract” in each category will be announced at the conclusion of the 45th Annual National SCDAA Convention. Special awards for the best student and trainee abstracts will also be given. We look forward to seeing you in October!

*Upon submission you will also be prompted to submit a Disclosure form.

Click here to submit your abstract today!

Click here to download and complete the Disclosure form.


The 11th Sickle Cell in Focus Conference

26-27 October 2017, Kingston, Jamaica

We are pleased to announce that Sickle Cell in Focus (SCiF) will be held for the first time in Kingston, Jamaica on October 26-27, 2017. This year, SCiF will be co-hosted by the National Heart, Lung, and Blood Institute and the University of West Indies, Jamaica. SCiF is a two-day, intensive, educational update on sickle cell disease. This year’s conference will focus on the latest clinical trials, the science and mechanisms for new therapeutic targets, and curative therapies. This two-day intensive educational conferences includes both clinical and scientific lectures, aimed at clinicians, academics, and other healthcare professionals involved in sickle cell disease around the world. Contact Rusinel Amarante| |