ライフサイエンスコーパス: sickle cell disease

1 rom 60 participants (20 controls and 40 with sickle cell disease).

2 ntage, as in the case of cystic fibrosis and sickle cell disease.

3 and offer prospects for curative therapy of sickle cell disease.

4 lly once daily) with placebo in persons with sickle cell disease.

5 ng the subcellular and cellular phenomena in sickle cell disease.

6 n a variety of diseases including cancer and sickle cell disease.

7 molytic diseases and conditions, sepsis, and sickle cell disease.

8 P-selectin, were evaluated in patients with sickle cell disease.

9 pt clinical trial in 12 subjects with stable sickle cell disease.

10 or a severe cerebral vasculopathy related to sickle cell disease.

11 on and acute lung injury in murine models of sickle cell disease.

12 increase muscle blood flow in patients with sickle cell disease.

13 nd doubled muscle perfusion in patients with sickle cell disease.

14 All 3 patients included had homozygous sickle cell disease.

15 cations among HPC transplant recipients with sickle cell disease.

16 ogenitor cell (HPC) transplantation can cure sickle cell disease.

17 bin (HbS) is the primary pathogenic event of sickle cell disease.

18 ar atrophy, cystic fibrosis, haemophilia and sickle cell disease.

19 emolytic anemia, anemia of inflammation, and sickle cell disease.

20 me excess alters the macrophage phenotype in sickle cell disease.

21 ads to formation of HbS (alpha2beta(S)2) and sickle cell disease.

22 or a clinical trial application for treating sickle cell disease.

23 eased in patients with autoimmune uveitis or sickle cell disease.

24 of the most important pathologic process in sickle cell disease.

25 ion and its pathophysiologic consequences in sickle cell disease.

26 rovide a unique view of the kidney injury in sickle cell disease.

27 maging of the perifoveal microvasculature in sickle cell disease.

28 Most children had either malaria or sickle cell disease.

29 comparative group of pregnant women without sickle cell disease.

30 end-organ damage and diminished survival in sickle cell disease.

31 adult beta-hemoglobin: beta-thalassemia and sickle cell disease.

32 ular vascular abnormalities in patients with sickle cell disease.

33 maternal and neonatal outcomes in women with sickle cell disease.

34 ne in black patients, with faster decline in sickle cell disease.

35 eneic hematopoietic cell transplantation for sickle cell disease.

36 n offers a favorable risk-benefit profile in sickle cell disease.

37 liable and accurate in newborn screening for sickle cell disease.

38 us thromboembolism observed in patients with sickle cell disease.

39 polymerization drives the pathophysiology of sickle cell disease.

40 and prevention of long-term complications of sickle cell disease.

41 vent liver injury in hemolytic diseases like sickle cell disease.

42 transplantation outcomes of in patients with sickle cell disease.

43 cause of acute lung disease in children with sickle-cell disease.

44 is a promising strategy for the treatment of sickle-cell disease.

45 rvation, 95% CI 40-86) than in those without sickle cell disease (2.4 per 1000 person-years of observ

46 sickle cell disease than in children without sickle cell disease (210 per 1000 person-years of observ

47 nty-one studies (including 26,349 women with sickle cell disease; 26,151,746 women without sickle cel

48 I 2.82-7.55), prematurity (4.33, 2.47-7.58), sickle cell disease (3.46, 1.63-7.37), immunosuppression

49 he pathophysiology and many subphenotypes of sickle cell disease; (3) clinical implications of person

50 onsecutive patients (3 men and 2 women) with sickle cell disease (4 patients with hemoglobin SS disea

51 Of the 55 babies and infants with confirmed sickle cell disease, 41 (75%) were enrolled into a progr

52 Mortality was higher in children with sickle cell disease (58 per 1000 person-years of observa

53 In sickle-cell disease, a point mutation in the beta-globin

54 In sickle cell disease, aberrant blood flow due to oxygen-d

55 Sickle-cell disease affects millions of individuals worl

56 natal health outcomes in pregnant women with sickle cell disease against a comparative group of pregn

57 adolescents with sickle cell disease aged 12-19 years, primary data on ad

58 g inflammation, cancer, neuroprotection, and sickle cell disease among many others.

59 olled 17 consecutive patients: 12 (71%) with sickle cell disease and 5 (29%) with beta-thalassaemia m

60 The beta-haemoglobinopathies, such as sickle cell disease and beta-thalassaemia, are caused by

61 in chain imbalance in erythroid progeny from sickle cell disease and beta-thalassemia patient-derived

62 oglobin (HbF) can ameliorate the severity of sickle cell disease and beta-thalassemia(1).

63 ion of fetal hemoglobin (HbF) in people with sickle cell disease and beta-thalassemia.

64 tical goal in the treatment of patients with sickle cell disease and beta-thalassemia.

65 dult erythrocytes can reduce the severity of sickle cell disease and beta-thalassemia.

66 Participants were tested for sickle cell disease and followed up for survival status

67 is simple risk score may guide patients with sickle cell disease and hematologists who are considerin

68 erentiating erythroid cells from people with sickle cell disease and in myeloma patients.

69 imilarities in the experience of living with sickle cell disease and living with other chronic illnes

70 ase that manifests clinical complications in sickle cell disease and other chronic hereditary or acqu

71 a from patients with known oxidative stress (sickle cell disease and sepsis) and from a patient with

72 lobinopathies or globin disorders, including sickle cell disease and thalassemia.

73 Of 996 patients with sickle cell disease and who underwent transplantation in

74 ardiovascular complications in patients with sickle-cell disease and discuss how screening and interv

75 Asthma is common in children with sickle-cell disease and is associated with increased inc

76 s increased about asthma as a comorbidity in sickle-cell disease and its effects on morbidity, substa

77 51 patients with sickle cell trait, 230 with sickle cell disease, and 8729 reference patients, with a

78 the antiphospholipid syndrome, preeclampsia, sickle cell disease, and biomaterial-induced thromboinfl

79 as hereditary hemochromatosis, thalassemia, sickle cell disease, and myelodysplasia that can lead to

80 in animal models of diseases such as cancer, sickle cell disease, and renal fibrosis.

81 Patients with sickle cell disease are at high risk for chronic hepatit

82 Sickle cell trait and sickle cell disease are thought to be independent risk f

83 globin disorders, including thalassaemia and sickle-cell disease, are the most common monogenic disea

84 ere, using vaso-occlusive episodes (VOEs) of sickle cell disease as a vascular disease model, we show

85 c nephropathy in European Americans and with sickle cell disease-associated nephropathy.

86 presented for immunisation were screened for sickle cell disease at five primary health-care centres

87 and the development of a device to identify sickle cell disease based on aqueous multiphase systems

88 ul for severe acute chest syndrome in adults sickle cell disease, because of the frequent hemodynamic

89 f 41), and 89.4% (42 of 47) of patients with sickle cell disease, beta-thalassemia, and hemophilia A/

90 babies per year are thought to be born with sickle cell disease, but accurate data are not available

91 ation occurs in hemolytic disorders, such as sickle cell disease, but the pathological relevance and

92 ian period 2: a missing beneficial factor in sickle cell disease by lowering pulmonary inflammation,

93 tion of the Glu6Val mutation responsible for sickle cell disease by using patient-derived stem and pr

94 Temporal macular involvement in sickle cell disease can now easily be detected by optica

95 a variety of important pathologies including sickle cell disease, cancer, inflammation, and fibrosis.

96 faster in patients with sickle cell trait or sickle cell disease compared with reference patients; it

97 We systematically screened for sickle cell disease consecutive newborn babies and infan

98 Sickle cell disease contributes substantially to mortali

99 In patients with sickle cell disease, crizanlizumab therapy resulted in a

100 QCT molecular counting to develop sgNIPTs of sickle cell disease, cystic fibrosis, spinal muscular at

101 free iron during hemolytic diseases such as sickle cell disease, dengue fever, malaria, and sepsis.

102 In Africa, 50-90% of children born with sickle cell disease die before they reach their fifth bi

103 In patients with sickle cell disease, donor and recipient red cell phenot

104 Living with a long-term condition such as sickle cell disease during adolescence constitutes a sig

105 The study population was all patients with sickle cell disease enrolled before March 31, 2015, in t

106 y be warranted when evaluating patients with sickle cell disease, even if asymptomatic with 20/20 vis

107 Three patients with sickle cell disease exhibiting preserved visual acuity b

108 Children with sickle cell disease experience organ damage, impaired qu

109 nts had beta-thalassaemia major, 27 (7%) had sickle cell disease, five (1%) had thalassodrepanocytosi

110 Although the molecular cause of sickle-cell disease has been known for more than half a

111 ger than 50 years, had genetically confirmed sickle cell disease (Hb SS) or sickle beta thalassemia (

112 est expression in children with both SMA and sickle cell disease (HbSS), corresponding with elevated

113 patient transition from paediatric to adult sickle cell disease health care is unlikely to address t

114 maternal and neonatal outcomes in women with sickle cell disease; however, the evidence stems from a

115 ort the inclusion of neurological disorders, sickle cell disease, immunosuppression, diabetes, and ag

116 Sickle cell disease impacts on multiple facets of an ado

117 rations made in a single referral center for sickle cell disease in 2016.

118 We enrolled patients with sickle cell disease in a single-center, open-label pilot

119 Increased longevity of patients with sickle cell disease in high-income, middle-income, and l

120 onducted to quantify the association between sickle cell disease in pregnancy and adverse maternal an

121 udy bodes well for the care of patients with sickle cell disease in resource-poor countries.

122 d mortality were high in young children with sickle cell disease in this Kenyan cohort, both were red

123 have reduced the morbidity and mortality of sickle-cell disease in developed countries.

124 ess is thought to contribute to pathology in sickle cell disease, in this issue of Blood, Morris et a

125 will help to inform national strategies for sickle cell disease, including neonatal screening.

126 Microvascular abnormalities in sickle cell disease involved both the superficial and th

127 Sickle cell disease is a common and life-threatening hae

128 Sickle cell disease is a life-threatening inherited cond

129 Pregnancy in women with sickle cell disease is associated with adverse maternal

130 The pathology of sickle cell disease is caused by polymerization of the a

131 The majority of morbidity and mortality in sickle cell disease is caused by vaso-occlusion: circula

132 Sickle cell disease is characterized by hemolytic anemia

133 Sickle cell disease is fundamentally a kinetic disorder,

134 Sickle cell disease is highly prevalent in sub-Saharan A

135 Sickle cell disease is one of the most common, life-thre

136 own for more than 60 years that the cause of sickle cell disease is polymerization of a hemoglobin mu

137 Sickle Cell Disease is the commonest monogenic haemoglob

138 Sickle cell disease is the most common severe monogenic

139 Although the root cause of sickle cell disease is the polymerization of hemoglobin

140 The burden of sickle-cell disease is expected to continue to rise over

141 from microcirculatory impairment, including sickle cell disease, ischemic heart disease, and heart f

142 Patients with sickle cell disease may develop various macular vascular

143 Temporal macular atrophy in sickle cell disease may have direct consequences on visu

144 Among children with sickle cell disease, mortality was lower in those who en

145 Patients with sickle cell disease (n = 17) and malaria (n = 15) contri

146 Despite advances in the management of sickle cell disease, obstetrics, and neonatal medicine,

147 e KGBCS, and 128 (0.8%) of these infants had sickle cell disease, of whom 70 (54.7%) enrolled at the

148 one marrow transplantation for patients with sickle cell disease on a clinical trial that had a compa

149 children, pregnant women, and patients with sickle cell disease or AIDS.

150 eme in pregnant women either due to malaria, sickle cell disease or other hemolytic diseases, will en

151 th HIV or malaria, or who are compromised by sickle cell disease or severe malnutrition.

152 flammation-related organ damage in models of sickle-cell disease or endotoxin-induced septic shock.

153 troversies include the role of haemolysis in sickle cell disease pathophysiology, optimal management

154 Our study shows that outcome is impaired in sickle cell disease patients receiving extracorporeal li

155 essel density was significantly lower in the sickle cell disease patients than in the control group i

156 avascular zone) was significantly larger in sickle cell disease patients than in the control group,

157 from healthy donors and thrombocytopenic and sickle cell disease patients.

158 ren (aged 6-60 months), most with malaria or sickle cell disease, presenting February 2013 through Ma

159 the hemoglobinopathies (beta-thalassemia and sickle cell disease); rare genetic disorders of RBC prod

160 Over the 8-year period, 22 patients with sickle cell disease required extracorporeal life support

161 ew byproducts, the primary genetic causes of sickle cell disease (requiring a transversion in HBB) an

162 ion of fetal haemoglobin to the forefront of sickle-cell disease research.

163 e uveitis, systemic lupus erythematosus, and sickle cell disease, respectively, as well as in 41 age-

164 Sickle cell disease results from a homozygous missense m

165 e (ACS) is a common, serious complication of sickle cell disease (SCD) and a leading cause of hospita

166 Pain is a life-long symptom in sickle cell disease (SCD) and a predictor of disease pro

167 ameliorate the severe beta-globin disorders sickle cell disease (SCD) and beta-thalassemia by induct

168 ene (HBB; which encodes beta-globin), mainly sickle cell disease (SCD) and beta-thalassemia, become s

169 fully applied to hemoglobinopathies, such as sickle cell disease (SCD) and beta-thalassemia.

170 tment for beta-hemoglobinopathies, including sickle cell disease (SCD) and beta-thalassemia.

171 ta2) is an important therapeutic approach in sickle cell disease (SCD) and beta-thalassemia.

172 erythrocyte SphK1 activity is upregulated in sickle cell disease (SCD) and contributes to sickling an

173 eness (AHR) affects 55%-77% of children with sickle cell disease (SCD) and occurs even in the absence

174 hemopexin deficiency, and kidney function in sickle cell disease (SCD) and report that (1) acute elev

175 Sickle cell disease (SCD) and thalassemias (Thal) are co

176 Human immunodeficiency virus (HIV) and sickle cell disease (SCD) are regarded as endemic in ove

177 sfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases

178 beta-Thalassemia and sickle cell disease (SCD) are the most prevalent monogen

179 and high counts of circulating HSC/P seen in sickle cell disease (SCD) as a result of vascular damage

180 -independent EPO regulation, we assessed two sickle cell disease (SCD) cohorts for genetic associatio

181 Sickle cell disease (SCD) complications are associated w

182 n two patients admitted to the hospital with sickle cell disease (SCD) crisis.

183 issue of Blood, Matte et al demonstrate that sickle cell disease (SCD) disrupts inflammation-resoluti

184 Persons with sickle cell disease (SCD) exhibit subjective hypersensit

185 Children with sickle cell disease (SCD) experience cognitive deficits

186 CRISPR/Cas enhanced correction of the sickle cell disease (SCD) genetic defect in patient-spec

187 ce the frequency of vaso-occlusive episodes, sickle cell disease (SCD) has continued to be treated pr

188 Patients with sickle cell disease (SCD) have markers of chronic inflam

189 globin expression and the pathophysiology of sickle cell disease (SCD) in a NRF2 knockout (SCD/NRF2(-

190 and sustainability of newborn screening for sickle cell disease (SCD) in sub-Saharan Africa and othe

191 Sickle cell disease (SCD) is a genetic disorder caused b

192 Sickle cell disease (SCD) is a genetic disorder that pos

193 Sickle cell disease (SCD) is a genetic hemoglobinopathy

194 Sickle cell disease (SCD) is a hematological disorder le

195 Sickle cell disease (SCD) is a highly complex genetic bl

196 Sickle cell disease (SCD) is a major global health conce

197 Sickle cell disease (SCD) is a monogenic disorder that a

198 Sickle cell disease (SCD) is a severe genetic blood diso

199 Sickle cell disease (SCD) is a worldwide distributed her

200 Sickle cell disease (SCD) is a worldwide hematological d

201 Sickle cell disease (SCD) is associated with chronic act

202 Pulmonary hypertension (PH) in adults with sickle cell disease (SCD) is associated with early morta

203 Sickle cell disease (SCD) is caused by a variant hemoglo

204 Sickle cell disease (SCD) is caused by genetic defects i

205 Sickle cell disease (SCD) is characterized by a single p

206 entral nervous system (CNS) complications in sickle cell disease (SCD) is evolving.

207 ymptom of glomerular injury in patients with sickle cell disease (SCD) is microalbuminuria.

208 Sickle cell disease (SCD) is one of the most common here

209 Although a blood genetic disease, sickle cell disease (SCD) leads to a chronic vasculopath

210 Sickle cell disease (SCD) leads to significant morbidity

211 ective and more potent inhibitor, RN-1, in a sickle cell disease (SCD) mouse model.

212 nd colleagues demonstrate that patients with sickle cell disease (SCD) on hydroxyurea have lower cere

213 potheses relating intravascular hemolysis to sickle cell disease (SCD) pathogenesis.

214 on of sickle hemoglobin (HbS) play a role in sickle cell disease (SCD) pathogenesis.

215 gh hemoglobin-hyperviscous" subphenotypes of sickle cell disease (SCD) patients is based on North Ame

216 ating MPs on endothelial cells (ECs) from 60 sickle cell disease (SCD) patients.

217 oses reduces platelet adhesion but increases sickle cell disease (SCD) red blood cell (RBC) adhesion

218 Effective medical management for sickle cell disease (SCD) remains elusive.

219 of vaso-occlusive crises (VOC) or events in sickle cell disease (SCD) remains limited to symptom rel

220 Sickle cell disease (SCD) results from a hemoglobin (Hb)

221 Sickle cell disease (SCD) results in cardiopulmonary dys

222 Sickle cell disease (SCD) results in vascular occlusions

223 Patients with sickle cell disease (SCD) suffer from intense pain that

224 ) induction is a well-validated strategy for sickle cell disease (SCD) treatment.

225 ll-free heme in plasma from 47 patients with sickle cell disease (SCD) was sequestered in circulating

226 openia are common clinical manifestations of sickle cell disease (SCD) with unclear mechanisms.

227 Pain is a characteristic feature of sickle cell disease (SCD), 1 of the most common inherite

228 Sickle cell disease (SCD), a congenital hemolytic anemia

229 Sickle cell disease (SCD), a genetic disorder of hemoglo

230 However, its function in sickle cell disease (SCD), a life-threatening hemolytic

231 ythrocyte arachidonic acid (AA) in mice with sickle cell disease (SCD), a prevalent hemolytic genetic

232 In sickle cell disease (SCD), abnormal adhesion of RBCs to

233 pportive therapy to prevent complications of sickle cell disease (SCD), access to care is not univers

234 he proximate event in the pathophysiology of sickle cell disease (SCD), are needed to address the sev

235 nificant cause of morbidity and mortality in sickle cell disease (SCD), but preventive, diagnostic, a

236 ingosine 1-phosphate (S1P) is detrimental in Sickle Cell Disease (SCD), but the mechanistic basis rem

237 prophylaxis is recommended for persons with sickle cell disease (SCD), but the value of this has bee

238 Altered mitochondrial function occurs in sickle cell disease (SCD), due in part to low nitric oxi

239 Certain populations, including people with sickle cell disease (SCD), exhibit a greater prevalence

240 ould benefit chronic pain conditions such as sickle cell disease (SCD), for which patients may requir

241 hich leads to the systemic manifestations of sickle cell disease (SCD), including vaso-occlusion, ana

242 Hydroxyurea (HU), a common therapy for sickle cell disease (SCD), induces fetal Hb production a

243 cts approximately 10% of adult patients with sickle cell disease (SCD), particularly those with the h

244 n these genes cause cystic fibrosis (CF) and sickle cell disease (SCD), respectively.

245 tients after splenectomy or in patients with sickle cell disease (SCD), the number of circulating red

246 ts with hematologic disorders, most commonly sickle cell disease (SCD), there is significant concern

247 In sickle cell disease (SCD), treatment of recurrent vasooc

248 Sickle cell disease (SCD)-associated nephropathy is a ma

249 sis (VOC) is the most common complication of sickle cell disease (SCD).

250 reportedly occurs in 10% of adolescents with sickle cell disease (SCD).

251 dhesion, correlate with clinical severity in sickle cell disease (SCD).

252 ies have hampered their development to treat sickle cell disease (SCD).

253 ons are of vital importance in patients with sickle cell disease (SCD).

254 Cs) are in clinical trials for patients with sickle cell disease (SCD).

255 helin-1 (ET-1), a potent vasoconstrictor, in sickle cell disease (SCD).

256 een a long-standing goal in the treatment of sickle cell disease (SCD).

257 onary hypertension has been controversial in sickle cell disease (SCD).

258 , and survival advantage in a mouse model of sickle cell disease (SCD).

259 processes modulating the pathophysiology of sickle cell disease (SCD).

260 ular event that leads to hemolytic anemia in sickle cell disease (SCD).

261 diate vaso-occlusive events in patients with sickle cell disease (SCD).

262 a major cause of morbidity and mortality in sickle cell disease (SCD).

263 ion and injury, and promote vasoocclusion in sickle cell disease (SCD).

264 cations for efforts to prevent thrombosis in sickle cell disease (SCD).

265 revalent and debilitating hemolytic disorder sickle cell disease (SCD).

266 nts in the microvasculature of patients with sickle cell disease (SCD).

267 injury (AKI) is a major clinical concern in sickle cell disease (SCD).

268 ctivity correlates with heme levels in mouse sickle cell disease (SCD).

269 worsens symptoms and crises in patients with sickle cell disease (SCD).

270 evaluated for hemoglobin disorders including sickle cell disease (SCD).

271 lowering may also have beneficial effects in sickle cell disease (SCD).

272 ry cause of morbidity and hospitalization in sickle cell disease (SCD); however, only 4 therapies (hy

273 mergent complication affecting patients with sickle cell disease (SCD); however, the molecular mechan

274 ed to test the feasibility of implementing a sickle cell disease screening programme using innovative

275 res et Drepanocytose, ie, Heart Arteries and Sickle Cell Disease) study prospectively recruited pedia

276 In several genetic conditions, including sickle cell disease, thalassemia, and G6PD deficiency, e

277 y disorders of erythrocyte hydration include sickle cell disease, thalassemia, hemoglobin CC, and her

278 ities in RBCs, including altered hematocrit, sickle cell disease, thalassemia, hemolytic anemias, and

279 to hospital was also higher in children with sickle cell disease than in children without sickle cell

280 clined significantly faster in patients with sickle cell disease than in patients with sickle cell tr

281 pport the design of innovative therapies for sickle-cell disease that are based on fetal haemoglobin.

282 literature about how adolescent's experience sickle cell disease, this body of research has not been

283 For adolescents with sickle cell disease to be cared for and supported approp

284 mutant mice with a humanized mouse model of sickle cell disease to directly explore the relevance of

285 ring adolescents' experiences of living with sickle cell disease to make recommendations for practice

286 rities to consider for any young person with sickle cell disease transitioning from paediatric to adu

287 disease and sepsis) and from a patient with sickle cell disease treated with the antioxidant N-acety

288 controlled trial involving participants with sickle cell disease, voxelotor significantly increased h

289 Sickle cell disease was less common in children older th

290 n for admission to hospital among those with sickle cell disease was severe anaemia (incidence 48 per

291 h SCA, followed in the Cooperative Study for Sickle Cell Disease, was constructed using the first pul

292 years) with electrophoretic confirmation of sickle cell disease were included and analyzed.

293 Children with sickle cell disease were offered confirmatory testing an

294 Clinical manifestations of sickle cell disease were reduced or absent during the fo

295 so shows that neutrophils from patients with sickle cell disease were unresponsive to one of two majo

296 ickle cell disease; 26,151,746 women without sickle cell disease) were eligible for inclusion.

297 h HbSS genotype, compared with women without sickle cell disease, were at increased risk of maternal

298 t-free survival is improved in patients with sickle cell disease who receive an allogenic transplanta

299 d between birth and 5 years with and without sickle cell disease, who were resident within the Kilifi

300 assess the visual function of patients with sickle cell disease with no visual symptoms despite temp