ライフサイエンスコーパス: PNH

1 PNH has been considered a monogenic disease that results

2 PNH III erythrocytes circulate 6 to 60 days in vivo.

3 PNH is due to the expansion of a cell clone that has acq

4 PNH neutrophils (CD15(+)/CD66b(-)/CD16(-)) were detected

5 PNH red blood cells also were identified at a frequency

6 PNH T cells comprise mainly naive cells (CD45RA(+)CD45R0

7 PNH type III cells were completely resistant to aerolysi

8 background in the TCR repertoire of 6 of 11 PNH patients.

9 targeted deep sequencing of an additional 36 PNH patients.

10 is of natural killer (NK) cell subsets in 47 PNH patients revealed that the ratio of CD56(bright):CD5

11 Platelet expression of PrPc was studied in 8 PNH patients.

12 We present data from a PNH patient in remission for 20 years who still had sign

13 ied as determinate versus indeterminate in a PNH-mediated step.

14 ypothesis was considered that expansion of a PNH clone may be a marker of immune-mediated disease and

15 herapy; in myelodysplasia, the presence of a PNH population was strongly correlated with hematologic

16 For patients with a PNH clone (defined by the presence of GPI-AP-deficient g

17 requent in AA/PNH (56%) than in AA without a PNH clone (37%; z = 3.36).

18 nosis in patients with otherwise typical AA, PNH, and MDS.

19 PNH associated with bone marrow failure (AA/PNH and MDS/PNH).

20 HLA-DR2 was more frequent in AA/PNH (56%) than in AA without a PNH clone (37%; z = 3.36)

21 ures of 60 patients presenting with acquired PNH.

22 and axis determinacy are likely to be among PNH targets.

23 reviously detected in CD34 cells from AA and PNH patients reveals the presence of many similarities t

24 esembles the coexistence of normal cells and PNH cells in patients with an established PNH clone.

25 Caspr2 is an autoantigen of encephalitis and PNH previously attributed to VGKC antibodies.

26 effect of HSC-specific T cells on normal and PNH HSCs.

27 ed whole-exome sequencing of paired PNH+ and PNH- fractions on samples taken from 12 patients as well

28 ws consistent phenotypic differences between PNH B cells and residual normal B cells.

29 zumab and to define the relationship between PNH and bone marrow failure syndromes.

30 In view of the close relationship between PNH and idiopathic aplastic anemia (IAA), it has been su

31 We identified 30 patients as having both PNH and PMG on brain imaging, reviewed clinical data and

32 acy study evaluating eculizumab in a broader PNH patient population.

33 It was characterized by PNH in the trigones, temporal and posterior horns of the

34 It was characterized by PNH lining the lateral body and frontal horns of the lat

35 ic change is an additional feature shared by PNH and aplastic anemia.

36 c germ-line PIGA mutations that do not cause PNH have been shown to be responsible for a condition kn

37 s; however, alternative mutations that cause PNH have recently been discovered.

38 mily, is similar to mutations known to cause PNH as a result of PIGA dysfunction, and was absent in 4

39 one to a size sufficient to produce clinical PNH is not observed.

40 studies suggest that patients with clinical PNH who are treated with eculizumab have a benign clinic

41 glycosylphosphatidylinositol (GPI)-deficient PNH cells.

42 y, the use of aerolysin allowed us to detect PNH populations that could not be detected by standard f

43 Insights into the relevance of detecting PNH cells in PNH and other bone marrow failure disorders

44 conduction velocity, db/db mice also develop PNH.

45 protein expression at presentation developed PNH after therapy (n = 16).

46 rythrophagocytosis in samples from different PNH patients correlated well with the individual level o

47 he majority of patients with active disease, PNH B cells comprised mainly naive cells with a CD27(-)I

48 s not sufficient to cause the human disease, PNH.

49 rfaces of the blade, indicating that ectopic PNH can cause changes in cell division rates.

50 l types of autoimmune or viral encephalitis, PNH, or mutations of the Caspr2-encoding gene.

51 was performed on patients with encephalitis, PNH, or both.

52 nd PNH cells in patients with an established PNH clone.

53 pointed; it has been suggested that existing PNH clones may have a conditional growth advantage depen

54 erlecan in these functions could account for PNH when perlecan is lacking.

55 To develop a gene therapy approach for PNH, a retroviral vector construct, termed MPIN, was mad

56 n allows for a simple, inexpensive assay for PNH that is sensitive and specific.

57 ow transplantation remains the only cure for PNH but should be reserved for patients with suboptimal

58 nd a shared pathophysiological mechanism for PNH and PMG.

59 nding may guide future treatment options for PNH but also has potential implications for the descript

60 ded the nerve trunk as the original site for PNH.

61 rs after the description of the Ham test for PNH, Dacie commented that "the essential basis of the di

62 pathophysiology, diagnosis, and therapy for PNH, aHUS, and CAD.

63 splantation is the only curative therapy for PNH.

64 e have investigated this possibility in four PNH patients by treating them with granulocyte colony-st

65 We found that CD59(-)CD34(+) cells from PNH patients proliferated to levels approaching those of

66 I(-) B cells in 2 patients in remission from PNH suggest that the life span of a B-cell clone can be

67 hematopoiesis, that is, a large granulocyte PNH clone, the residual normal B cells had a predominant

68 mia of paroxysmal nocturnal haemoglobinuria (PNH).

69 Patients with bone marrow failure who have PNH cells detected by high-sensitivity flow cytometry ha

70 ts with paroxysmal nocturnal hemoglobinuria (PNH) (18 with active disease and 1 spontaneous remitter)

71 such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

72 ment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome, blocks the

73 ions of paroxysmal nocturnal hemoglobinuria (PNH) and improve quality of life and overall survival, b

74 ases of paroxysmal nocturnal hemoglobinuria (PNH) and some myeloproliferative neoplasms (MPNs), and r

75 tory of paroxysmal nocturnal hemoglobinuria (PNH) and to review new therapeutic strategies for contro

76 AA and paroxysmal nocturnal hemoglobinuria (PNH) are related clinically, and glycophosphoinositol (G

77 Paroxysmal nocturnal hemoglobinuria (PNH) arises from a somatic mutation of the PIG-A gene in

78 ence of paroxysmal nocturnal hemoglobinuria (PNH) at diagnosis, with no substantial change in the ove

79 is of a paroxysmal nocturnal hemoglobinuria (PNH) case without somatic mutations in PIGA, we performe

80 Paroxysmal nocturnal hemoglobinuria (PNH) cells are partially (type II) or completely (type I

81 Paroxysmal nocturnal hemoglobinuria (PNH) cells are susceptible to hemolysis because of a los

82 ed that paroxysmal nocturnal hemoglobinuria (PNH) cells may proliferate through their intrinsic resis

83 ts with paroxysmal nocturnal hemoglobinuria (PNH) comprise a mixture of residual normal and glycosylp

84 ts with paroxysmal nocturnal hemoglobinuria (PNH) comprise variable mixtures of normal B cells produc

85 way, of paroxysmal nocturnal hemoglobinuria (PNH) erythrocytes in human serum.

86 erlying paroxysmal nocturnal hemoglobinuria (PNH) has been shown to reside in PIGA, a gene that encod

87 ts with paroxysmal nocturnal hemoglobinuria (PNH) have blood cells deficient in glycosyl phosphatidyl

88 Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell disorder charac

89 Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal stem cell disorder caused by a somatic

90 Paroxysmal nocturnal hemoglobinuria (PNH) is a disorder of hematopoietic stem cells that has

91 Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal disease of hematopoietic s

92 Paroxysmal nocturnal hemoglobinuria (PNH) is a rare bone marrow failure disorder that manifes

93 Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal blood disorder that manifests with

94 Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of the hematopoietic

95 Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic disorder with i

96 Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem-cell disorder in

97 Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia characterized by th

98 Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired stem cell disorder characterized by

99 Paroxysmal nocturnal hemoglobinuria (PNH) is caused by phosphatidylinositol glycan-class A (P

100 Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by complement-mediated intravascul

101 Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by complement-mediated intravascul

102 Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis, which

103 Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by the presence in the patient's h

104 mark of paroxysmal nocturnal hemoglobinuria (PNH) is chronic intravascular hemolysis that is a conseq

105 yndrome paroxysmal nocturnal hemoglobinuria (PNH) is intimately related to aplastic anemia because ma

106 BMF) in paroxysmal nocturnal hemoglobinuria (PNH) is not yet known.

107 ture of paroxysmal nocturnal hemoglobinuria (PNH) is that in each patient glycosylphosphatidylinosito

108 tion of paroxysmal nocturnal hemoglobinuria (PNH) is thrombosis.

109 ts with paroxysmal nocturnal hemoglobinuria (PNH) lack GPI proteins on the surface of somatically mut

110 MDS) or paroxysmal nocturnal hemoglobinuria (PNH) occurring as a late complication of AA, we studied

111 vity in paroxysmal nocturnal hemoglobinuria (PNH) patients on eculizumab treatment.

112 and 21 paroxysmal nocturnal hemoglobinuria (PNH) patients, in whom specific CDR3 sequences and clona

113 ytes of paroxysmal nocturnal hemoglobinuria (PNH) patients, who suffer from complement-mediated hemol

114 ts with paroxysmal nocturnal hemoglobinuria (PNH) undergoing eculizumab treatment, which are opsonize

115 nt with paroxysmal nocturnal hemoglobinuria (PNH) who does not have a mutation of PIG-A, but in whom

116 ysis in paroxysmal nocturnal hemoglobinuria (PNH), a disease that manifests after clonal expansion of

117 ment of paroxysmal nocturnal hemoglobinuria (PNH), a rare but life-threatening hematologic disease, h

118 In paroxysmal nocturnal hemoglobinuria (PNH), acquired somatic mutations in the PIG-A gene give

119 In paroxysmal nocturnal hemoglobinuria (PNH), an acquired mutation of the PIGA gene results in t

120 a (AA), paroxysmal nocturnal hemoglobinuria (PNH), and myelodysplasia (MDS).

121 a (AA), paroxysmal nocturnal hemoglobinuria (PNH), and some forms of myelodysplasia (MDS).

122 In paroxysmal nocturnal hemoglobinuria (PNH), hematopoietic cells lacking glycosylphosphatidylin

123 ts with paroxysmal nocturnal hemoglobinuria (PNH), leading to deficiency of GPI-linked proteins on th

124 disease paroxysmal nocturnal hemoglobinuria (PNH), somatic mutations result in a deficiency of glycos

125 ts with paroxysmal nocturnal hemoglobinuria (PNH).

126 ts with paroxysmal nocturnal hemoglobinuria (PNH).

127 ecially paroxysmal nocturnal hemoglobinuria (PNH).

128 ts with paroxysmal nocturnal hemoglobinuria (PNH); the authors demonstrate that these agents reach th

129 f HLA-DR2 was found in all frankly hemolytic PNH and in PNH associated with bone marrow failure (AA/P

130 In 10 patients with hemolytic PNH the telomeres in sorted GPI- granulocytes were short

131 njection given to 22 patients with hemolytic PNH while receiving eculizumab therapy.

132 rs with periventricular nodular heterotopia (PNH) are etiologically heterogeneous, and their genetic

133 MG) and periventricular nodular heterotopia (PNH) are two developmental brain malformations that have

134 Peripheral nerve hyperexcitability (PNH) is one of the distal peripheral neuropathy phenotyp

135 ngenital peripheral nerve hyperexcitability (PNH) is usually associated with impaired function of vol

136 eralized peripheral nerve hyperexcitability (PNH).

137 With a goal of improving PNH therapy, we characterized the activity of anti-C3b/i

138 In PNH patients with predominantly GPI-deficient hematopoie

139 In PNH, an entire class of proteins is not displayed on the

140 T cells were significantly more abundant in PNH patients than in healthy controls; their reactivity

141 as found in all frankly hemolytic PNH and in PNH associated with bone marrow failure (AA/PNH and MDS/

142 s directly confer resistance to apoptosis in PNH.

143 Because in PNH the biosynthesis of the glycolipid molecule glycosyl

144 H patients and may be responsible for BMF in PNH.

145 into the relevance of detecting PNH cells in PNH and other bone marrow failure disorders are highligh

146 gest that expansion of PIGA-mutated cells in PNH marrow is due to a growth defect in nonmutated cells

147 persensitivity to complement of red cells in PNH patients, manifested by intravascular hemolysis.

148 ts for the occurrence of spontaneous cure in PNH, consequent on clonal extinction.

149 of this hypothesis, we have demonstrated in PNH patients the presence of CD8(+) T cells reactive aga

150 pharmacodynamic (PD) study of eculizumab in PNH patients which shows that CH50 is a promising biomar

151 ion with eculizumab has a dramatic effect in PNH and has a major impact in the prevention of thrombos

152 Why PIGA-mutated cells are able to expand in PNH marrow, however, is as yet unclear.

153 an invariant TCRalpha chain, is expanded in PNH patients and may be responsible for BMF in PNH.

154 and may account for the clonal expansion in PNH and MPNs and in gene therapy patients after vector i

155 ays necessary to explain clonal expansion in PNH.

156 ecting a component of bone marrow failure in PNH.

157 A mutations have relevance to those found in PNH.

158 ns, accessory genetic events are frequent in PNH, suggesting a stepwise clonal evolution derived from

159 tein 5, stops the intravascular hemolysis in PNH.

160 d that the PIG-A gene may be hypermutable in PNH.

161 viding a potential new therapeutic option in PNH.

162 of heterologous complement activation or in PNH patients, impaired erythrocyte DAF activity and enha

163 e phenotype of GPI-deficient T cells seen in PNH patients with active disease likely reflects the phe

164 ow failure that is almost invariably seen in PNH.

165 s of these adaptive NK cells were similar in PNH patients and healthy individuals.

166 ife, and reducing the risk for thrombosis in PNH patients.

167 ding of the pathophysiology of thrombosis in PNH, as well as the treatment of thrombosis, will be dis

168 some of the complexity of the thrombosis in PNH.

169 sent it to the members of the International PNH Interest Group and to the physicians participating i

170 hysicians participating in the International PNH Registry.

171 eizures, 5 neuropathy or PNH, and 1 isolated PNH.

172 lizumab levels may help physicians to manage PNH patients receiving eculizumab.

173 PIGA mutations have been identified in many PNH patients, it has been proposed that germline mutatio

174 ted with bone marrow failure (AA/PNH and MDS/PNH).

175 ondition leads to potassium channel-mediated PNH, thus identifying them as a potential drug target to

176 proposed that distinguishes immune-mediated PNH (irrespective of whether VGKC antibodies are detecta

177 More strikingly, moving PNH expression from the central to the peripheral domain

178 NH stem cell, absolute numbers of both naive PNH CD4(+) cells and CD8(+) cells show an inverse correl

179 wever, 6H4 detected PrPc on Western blots of PNH platelets, demonstrating that the lack of 6H4 bindin

180 the characteristic and pathogenetic cause of PNH.

181 ab dramatically alters the natural course of PNH, reducing symptoms and disease complications as well

182 ns are not sufficient for the development of PNH.

183 am test and permits concomitant diagnosis of PNH in about 20% of patients with myelodysplasia (a rate

184 immune) present in the marrow environment of PNH patients.

185 , all 5 patients survive without evidence of PNH 5 to 39 months after transplantation.

186 marrow failure who have clinical evidence of PNH at presentation will require PNH-specific therapy.

187 Evidence of PNH was found in 25 of 115 (22%) patients with aplastic

188 Here, we show that ectopic expression of PNH on the abaxial (lower) sides of lateral organs resul

189 insic growth advantage, but some features of PNH argue that there are intrinsic drivers of clonal exp

190 Schwartz-Jampel syndrome (SJS) is a form of PNH that is due to hypomorphic mutations of perlecan, th

191 by standard assays) from non-immune forms of PNH that include toxins, anterior horn cell degeneration

192 However, a considerable fraction of PNH patients show insufficient treatment response and re

193 mab inhibits the intravascular haemolysis of PNH, reduces transfusion requirements, stabilises haemog

194 chors; therefore, the phenotypic hallmark of PNH cells is an absence or marked deficiency of all GPI-

195 haemolysis that is the clinical hallmark of PNH is a consequence of deficiency of the complement inh

196 r and C3-mediated extravascular hemolysis of PNH erythrocytes and warrants consideration for the trea

197 bits in a dose-dependent manner hemolysis of PNH erythrocytes in a modified extended acidified serum

198 avascular and the intravascular hemolysis of PNH.

199 strategies for controlling the hemolysis of PNH.

200 The percentage of lysis of PNH cells after aerolysin exposure paralleled the percen

201 e for many of the clinical manifestations of PNH.

202 e for patients with severe manifestations of PNH.

203 ss central to the morbidity and mortality of PNH.

204 [PEG]-Cp40) on hemolysis and opsonization of PNH erythrocytes in an established in vitro system.

205 ely prevent hemolysis and C3 opsonization of PNH erythrocytes, and are excellent, and potentially cos

206 gene is not a factor in the pathogenesis of PNH.

207 tations are important in the pathogenesis of PNH.

208 For patients with a percentage of PNH cells that is below the threshold for producing labo

209 The mean percentage of PNH type III erythrocytes increased from 36.7 percent of

210 nts leads to recognition and phagocytosis of PNH erythrocytes by immune cells.

211 NH are applicable to a broader population of PNH patients than previously studied.

212 Resistance of PNH cells to aerolysin allows for a simple, inexpensive

213 , no evidence for a decreased sensitivity of PNH cells to T-cell-mediated immunity was observed.

214 ar and expression data showed sensitivity of PNH-associated mutants to proteasome degradation.

215 ndent predictors of response but the size of PNH clone did not correlate with improvement in blood co

216 a common pathogenesis in a wider spectrum of PNH syndromes.

217 warrants consideration for the treatment of PNH patients.

218 ss complement inhibitor for the treatment of PNH validates the concept of complement inhibition as an

219 d is in phase 2 development for treatment of PNH.

220 blood cell lysis correlated with the type of PNH erythrocytes.

221 Deficiency of CD59 on PNH red blood cells results in chronic complement-mediat

222 17 block both hemolysis and C3 deposition on PNH erythrocytes.

223 vestigate in an in vitro model the effect on PNH erythrocytes of a novel therapeutic strategy for mem

224 ntly prevented deposition of C3 fragments on PNH erythrocytes.

225 xamined the impact of alloimmune pressure on PNH and normal cells in the clinical setting of nonmyelo

226 from 79 patients with AA, Fanconi anemia, or PNH in comparison with normal controls.

227 encephalopathy or seizures, 5 neuropathy or PNH, and 1 isolated PNH.

228 vation that accounts for hemolysis and other PNH manifestations.

229 e performed whole-exome sequencing of paired PNH+ and PNH- fractions on samples taken from 12 patient

230 The frontal-perisylvian PNH-PMG subtype included eight patients (seven males and

231 PINHEAD (PNH) is expressed in the central domain of the developin

232 The posterior PNH-PMG subtype consisted of 20 patients (15 male and 5

233 ic syndrome (MDS), and patients with primary PNH.

234 evidence of PNH at presentation will require PNH-specific therapy.

235 sitivity toward complement and thus resemble PNH type II cells in patients with PNH.

236 Resting PNH (CD55(-)) platelets were devoid of surface PrPc, but

237 for GPI-anchored protein transfer for severe PNH.

238 Five patients with severe PNH underwent HCT from an HLA-matched family donor after

239 These in vitro and in vivo studies show PNH cells can be immunologically eradicated following no

240 Aerolysin should also be useful in studying PNH biology.

241 aboratory evidence of hemolysis (subclinical PNH), expansion of the clone to a size sufficient to pro

242 prevents C3 fragment deposition on surviving PNH erythrocytes.

243 de experimental support for the concept that PNH, like IAA, has an immune pathogenesis.

244 He concluded that PNH is a cell-intrinsic defect of erythrocytes that refl

245 extravascular hemolysis and demonstrate that PNH erythrocytes from anti-C5-treated patients are phago

246 ssue of the JCI, Shen et al. discovered that PNH is in fact a complex genetic disorder orchestrated b

247 We found that PNH blood cells (erythrocytes, lymphocytes, and granuloc

248 Our data indicate that PNH can occur even in the presence of fully assembled GP

249 Surprisingly, this work indicates that PNH has a clonal evolution and architecture strikingly s

250 Here we show that PNH II and III cells become highly susceptible to comple

251 n utero electroporation approach showed that PNH-related mutants and excess wild-type NEDD4L affect n

252 in Schwann cell physiology and suggest that PNH in SJS originates distally from synergistic actions

253 These observations suggest that PNH patients treated with eculizumab are left with clini

254 The PNH cells may have a selective advantage in resisting im

255 The PNH-PMG subtypes described here have distinct imaging an

256 Both the presence of HLA-DR2 and the PNH clone were independent predictors of response but th

257 tol (GPI)-deficient B cells derived from the PNH hematopoietic stem cell.

258 topoiesis was predominantly derived from the PNH stem cell, absolute numbers of both naive PNH CD4(+)

259 , we now have identified cells that have the PNH phenotype, at an average frequency of 22 per million

260 logical inhibitors, we demonstrated that the PNH is mediated by the decreased activity of K(v)1-chann

261 we explore a third possibility, whereby the PNH clone does not have a selective advantage.

262 rved; and in a significant proportion, their PNH erythrocytes become opsonized with complement C3.

263 eins serve as receptors for aerolysin; thus, PNH cells are resistant to aerolysin.

264 These findings suggest an approach to PNH treatment in which both intravascular and extravascu

265 y of TT30 derives from its direct binding to PNH erythrocytes; if binding to the erythrocytes is disr

266 ited the binding of both factors B and C3 to PNH and rabbit erythrocytes and blocked the ability of f

267 ic complications were rare and deaths due to PNH or complications of therapy were not observed.

268 n of the encoded E3 ubiquitin ligase lead to PNH associated with toe syndactyly, cleft palate and neu

269 er 50 years old, died of causes unrelated to PNH.

270 re highlighted, and indications for treating PNH patients with bone marrow transplantation and eculiz

271 entation class A [PIGA] gene) that underlies PNH.

272 Using PNH as a novel model with which to study B lymphopoiesis

273 e completely resistant to aerolysin, whereas PNH type II cells displayed intermediate sensitivity.

274 isruption of Dab1 and mTORC1 pathways, while PNH-related mutations are associated with deregulation o

275 marrow mononuclear cells from a patient with PNH, and myeloid/erythroid colonies and erythroid cells

276 e (TK-14(-)) established from a patient with PNH, as well as peripheral blood (PB) mononuclear cells

277 d (PB) mononuclear cells from a patient with PNH.

278 lated CD34+ progenitors from 4 patients with PNH and 27 controls.

279 cts of eculizumab treatment in patients with PNH are applicable to a broader population of PNH patien

280 cacy of eculizumab in pregnant patients with PNH by examining the birth and developmental records of

281 Aerolysin-resistant CFCs from patients with PNH exhibited clonal PIG-A mutations.

282 Eleven transfusion-dependent patients with PNH received infusions of eculizumab (600 mg) every week

283 ntly more frequently skewed in patients with PNH than in controls.

284 erize the long-term outcome of patients with PNH treated with eculizumab and to define the relationsh

285 creased by more than 2-fold in patients with PNH, compared with controls (28% +/- 19.6% vs 11.4% +/-

286 ss in the TCR BV repertoire of patients with PNH, compared with controls (R(2) values 0.82 vs 0.91, P

287 c of those classically seen in patients with PNH, including an increased sensitivity toward complemen

288 gene, we have found that in 5 patients with PNH, mu ranged from 1.24 x 10(-7) to 11.2 x 10(-7), agai

289 iled phenotypic analysis of 29 patients with PNH, this study shows consistent phenotypic differences

290 mab is not appropriate for all patients with PNH.

291 is safe and well tolerated in patients with PNH.

292 ment in the quality of life in patients with PNH.

293 lowing transfusions given to 6 patients with PNH.

294 inal complement components, in patients with PNH.

295 ed in mice may also operate in patients with PNH.

296 resemble PNH type II cells in patients with PNH.

297 to solicit data on pregnancies in women with PNH and sent it to the members of the International PNH

298 Eculizumab provided benefit for women with PNH during pregnancy, as evidenced by a high rate of fet

299 Data on 75 pregnancies in 61 women with PNH were evaluated.

300 the closely related protein PINHEAD/ZWILLE (PNH/ZLL).