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Items: 1 to 50 of 72

1.

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.

Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators.

N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14.

PMID:
31199090
2.

Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

Mistry PK, Balwani M, Baris HN, Turkia HB, Burrow TA, Charrow J, Cox GF, Danda S, Dragosky M, Drelichman G, El-Beshlawy A, Fraga C, Freisens S, Gaemers S, Hadjiev E, Kishnani PS, Lukina E, Maison-Blanche P, Martins AM, Pastores G, Petakov M, Peterschmitt MJ, Rosenbaum H, Rosenbloom B, Underhill LH, Cox TM.

Blood Cells Mol Dis. 2019 Jul;77:101-102. doi: 10.1016/j.bcmd.2019.04.003. Epub 2019 Apr 9. No abstract available.

PMID:
31029022
3.

Recent trends in treatment of thalassemia.

El-Beshlawy A, El-Ghamrawy M.

Blood Cells Mol Dis. 2019 May;76:53-58. doi: 10.1016/j.bcmd.2019.01.006. Epub 2019 Feb 4. Review.

PMID:
30792169
4.

Role of factor VIII-binding capacity of endogenous von Willebrand factor in the development of factor VIII inhibitors in patients with severe hemophilia A.

Repessé Y, Costa C, Palla R, Moshai EF, Borel-Derlon A, D'Oiron R, Rothschild C, El-Beshlawy A, Elalfy M, Ramanan V, Eshghi P, Oldenburg J, Pavlova A, Rosendaal FR, Peyvandi F, Kaveri SV, Lacroix-Desmazes S.

Haematologica. 2019 Aug;104(8):e369-e372. doi: 10.3324/haematol.2018.212001. Epub 2019 Jan 31. No abstract available.

5.

International sentinel site surveillance of patients with transfusional hemosiderosis treated with deferasirox in actual practice setting.

El-Beshlawy A, Inusa B, Beneitez Pastor D, Xicoy B, Soledada Duran Nieto M, Bruederle A, Azmon A, Gilotti G, Elalfy M.

Hematology. 2019 Dec;24(1):238-246. doi: 10.1080/16078454.2018.1558758.

PMID:
30558524
6.

Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

Mistry PK, Balwani M, Baris HN, Turkia HB, Burrow TA, Charrow J, Cox GF, Danda S, Dragosky M, Drelichman G, El-Beshlawy A, Fraga C, Freisens S, Gaemers S, Hadjiev E, Kishnani PS, Lukina E, Maison-Blanche P, Martins AM, Pastores G, Petakov M, Peterschmitt MJ, Rosenbaum H, Rosenbloom B, Underhill LH, Cox TM.

Blood Cells Mol Dis. 2018 Jul;71:71-74. doi: 10.1016/j.bcmd.2018.04.001. Epub 2018 Apr 9. No abstract available.

7.

Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5-year observational study (ENTRUST).

Vichinsky E, El-Beshlawy A, Al Zoebie A, Kamdem A, Koussa S, Chotsampancharoen T, Bruederle A, Gilotti G, Han J, Elalfy M.

Pediatr Blood Cancer. 2017 Sep;64(9). doi: 10.1002/pbc.26507. Epub 2017 Mar 10.

PMID:
28296163
8.

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.

Cox TM, Drelichman G, Cravo R, Balwani M, Burrow TA, Martins AM, Lukina E, Rosenbloom B, Goker-Alpan O, Watman N, El-Beshlawy A, Kishnani PS, Pedroso ML, Gaemers SJM, Tayag R, Peterschmitt MJ.

Blood. 2017 Apr 27;129(17):2375-2383. doi: 10.1182/blood-2016-12-758409. Epub 2017 Feb 6.

9.

Telomerase enzyme activity in Egyptian children with bone marrow failure and response to immunosuppressive therapy.

El Beshlawy A, Said F, El Ansary M, Hamdy M, Abdel-Azim K, Abdel-Razek AA, Abulata N, Abdel-Salam A.

Blood Cells Mol Dis. 2017 Mar;63:58-61. doi: 10.1016/j.bcmd.2017.01.012. Epub 2017 Jan 26. No abstract available.

PMID:
28160733
10.

Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.

El-Beshlawy A, Tylki-Szymanska A, Vellodi A, Belmatoug N, Grabowski GA, Kolodny EH, Batista JL, Cox GF, Mistry PK.

Mol Genet Metab. 2017 Jan - Feb;120(1-2):47-56. doi: 10.1016/j.ymgme.2016.12.001. Epub 2016 Dec 6.

11.

Limitations of serum ferritin to predict liver iron concentration responses to deferasirox therapy in patients with transfusion-dependent thalassaemia.

Porter JB, Elalfy M, Taher A, Aydinok Y, Lee SH, Sutcharitchan P, El-Ali A, Han J, El-Beshlawy A.

Eur J Haematol. 2017 Mar;98(3):280-288. doi: 10.1111/ejh.12830. Epub 2017 Jan 13.

PMID:
27859648
12.

A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.

Peyvandi F, Mannucci PM, Garagiola I, El-Beshlawy A, Elalfy M, Ramanan V, Eshghi P, Hanagavadi S, Varadarajan R, Karimi M, Manglani MV, Ross C, Young G, Seth T, Apte S, Nayak DM, Santagostino E, Mancuso ME, Sandoval Gonzalez AC, Mahlangu JN, Bonanad Boix S, Cerqueira M, Ewing NP, Male C, Owaidah T, Soto Arellano V, Kobrinsky NL, Majumdar S, Perez Garrido R, Sachdeva A, Simpson M, Thomas M, Zanon E, Antmen B, Kavakli K, Manco-Johnson MJ, Martinez M, Marzouka E, Mazzucconi MG, Neme D, Palomo Bravo A, Paredes Aguilera R, Prezotti A, Schmitt K, Wicklund BM, Zulfikar B, Rosendaal FR.

N Engl J Med. 2016 May 26;374(21):2054-64. doi: 10.1056/NEJMoa1516437.

13.

Erratum to: Response to hydroxycarbamide in pediatric β-thalassemia intermedia: 8 years' follow-up in Egypt.

El-Beshlawy A, El-Ghamrawy M, El-Ela MA, Said F, Adolf S, Abdel-Razek AR, Magdy RI, Abdel-Salam A.

Ann Hematol. 2016 Apr;95(5):851. doi: 10.1007/s00277-016-2635-9. No abstract available.

PMID:
26972109
14.

Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload.

Aydinok Y, Kattamis A, Cappellini MD, El-Beshlawy A, Origa R, Elalfy M, Kilinç Y, Perrotta S, Karakas Z, Viprakasit V, Habr D, Constantinovici N, Shen J, Porter JB; HYPERION Investigators.

Blood. 2015 Jun 18;125(25):3868-77. doi: 10.1182/blood-2014-07-586677. Epub 2015 May 1.

15.

Peripheral expression of hepcidin gene in Egyptian β-thalassemia major.

Aboul-Enein A, El-Beshlawy A, Hamdy M, Shaheen I, El-Saadany Z, Samir A, El-Samie HA.

Gene. 2015 Jun 15;564(2):206-9. doi: 10.1016/j.gene.2015.03.048. Epub 2015 Mar 25.

PMID:
25816754
16.

Prevalence and distribution of iron overload in patients with transfusion-dependent anemias differs across geographic regions: results from the CORDELIA study.

Aydinok Y, Porter JB, Piga A, Elalfy M, El-Beshlawy A, Kilinç Y, Viprakasit V, Yesilipek A, Habr D, Quebe-Fehling E, Pennell DJ.

Eur J Haematol. 2015 Sep;95(3):244-53. doi: 10.1111/ejh.12487. Epub 2015 Jan 8.

17.

Sustained improvements in myocardial T2* over 2 years in severely iron-overloaded patients with beta thalassemia major treated with deferasirox or deferoxamine.

Pennell DJ, Porter JB, Piga A, Lai YR, El-Beshlawy A, Elalfy M, Yesilipek A, Kilinç Y, Habr D, Musallam KM, Shen J, Aydinok Y; CORDELIA study investigators.

Am J Hematol. 2015 Feb;90(2):91-6. doi: 10.1002/ajh.23876. Epub 2014 Nov 19.

18.

Response to hydroxycarbamide in pediatric β-thalassemia intermedia: 8 years' follow-up in Egypt.

El-Beshlawy A, El-Ghamrawy M, EL-Ela MA, Said F, Adolf S, Abdel-Razek AR, Magdy RI, Abdel-Salam A.

Ann Hematol. 2014 Dec;93(12):2045-50. doi: 10.1007/s00277-014-2154-5. Epub 2014 Jul 27. Erratum in: Ann Hematol. 2016 Apr;95(5):851.

PMID:
25062719
19.

Serum ferritin level and morbidity risk in transfusion-independent patients with β-thalassemia intermedia: the ORIENT study.

Musallam KM, Cappellini MD, Daar S, Karimi M, El-Beshlawy A, Graziadei G, Magestro M, Wulff J, Pietri G, Taher AT.

Haematologica. 2014 Nov;99(11):e218-21. doi: 10.3324/haematol.2013.097220. Epub 2014 Jul 4. No abstract available.

20.

A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease.

Reid ME, El Beshlawy A, Inati A, Kutlar A, Abboud MR, Haynes J Jr, Ward R, Sharon B, Taher AT, Smith W, Manwani D, Ghalie RG.

Am J Hematol. 2014 Jul;89(7):709-13. doi: 10.1002/ajh.23725. Epub 2014 Apr 15.

21.

A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA).

Pennell DJ, Porter JB, Piga A, Lai Y, El-Beshlawy A, Belhoul KM, Elalfy M, Yesilipek A, Kilinç Y, Lawniczek T, Habr D, Weisskopf M, Zhang Y, Aydinok Y; CORDELIA study investigators.

Blood. 2014 Mar 6;123(10):1447-54. doi: 10.1182/blood-2013-04-497842. Epub 2014 Jan 2.

22.

Continuation of deferiprone therapy in patients with mild neutropenia may not lead to a more severe drop in neutrophil count.

El-Beshlawy AM, El-Alfy MS, Sari TT, Chan LL, Tricta F.

Eur J Haematol. 2014 Apr;92(4):337-40. doi: 10.1111/ejh.12241. Epub 2014 Jan 30.

PMID:
24330079
23.

Low prevalence of cardiac siderosis in heavily iron loaded Egyptian thalassemia major patients.

El Beshlawy A, El Tagui M, Hamdy M, El Ghamrawy M, Azim KA, Salem D, Said F, Samir A, St Pierre T, Pennell DJ.

Ann Hematol. 2014 Mar;93(3):375-9. doi: 10.1007/s00277-013-1876-0. Epub 2013 Aug 15.

PMID:
23949317
24.

A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease.

Kutlar A, Reid ME, Inati A, Taher AT, Abboud MR, El-Beshlawy A, Buchanan GR, Smith H, Ataga KI, Perrine SP, Ghalie RG.

Am J Hematol. 2013 Nov;88(11):E255-60. doi: 10.1002/ajh.23533. Epub 2013 Oct 3.

25.

Multicenter validation of spin-density projection-assisted R2-MRI for the noninvasive measurement of liver iron concentration.

St Pierre TG, El-Beshlawy A, Elalfy M, Al Jefri A, Al Zir K, Daar S, Habr D, Kriemler-Krahn U, Taher A.

Magn Reson Med. 2014 Jun;71(6):2215-23. doi: 10.1002/mrm.24854. Epub 2013 Jul 2.

26.

Intrafamilial transmission of hepatitis C infection in Egyptian multitransfused thalassemia patients.

Said F, El Beshlawy A, Hamdy M, El Raziky M, Sherif M, Abdel kader A, Ragab L.

J Trop Pediatr. 2013 Aug;59(4):309-13. doi: 10.1093/tropej/fmt017. Epub 2013 Mar 28.

PMID:
23542535
27.

Study of serum hepcidin in hereditary hemolytic anemias.

El Beshlawy A, Alaraby I, Abdel Kader MS, Ahmed DH, Abdelrahman HE.

Hemoglobin. 2012;36(6):555-70. doi: 10.3109/03630269.2012.721151. Epub 2012 Oct 23.

PMID:
23088733
28.

Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy.

Porter JB, Elalfy MS, Taher AT, Aydinok Y, Chan LL, Lee SH, Sutcharitchan P, Habr D, Martin N, El-Beshlawy A.

Ann Hematol. 2013 Jan;92(2):211-9. doi: 10.1007/s00277-012-1588-x. Epub 2012 Oct 21.

29.

Revised recommendations for the management of Gaucher disease in children.

Kaplan P, Baris H, De Meirleir L, Di Rocco M, El-Beshlawy A, Huemer M, Martins AM, Nascu I, Rohrbach M, Steinbach L, Cohen IJ.

Eur J Pediatr. 2013 Apr;172(4):447-58. doi: 10.1007/s00431-012-1771-z. Epub 2012 Jul 8. Review.

PMID:
22772880
30.

Prenatal diagnosis for thalassaemia in Egypt: what changed parents' attitude?

El-Beshlawy A, El-Shekha A, Momtaz M, Said F, Hamdy M, Osman O, Meshaal S, Gafaar T, Petrou M.

Prenat Diagn. 2012 Aug;32(8):777-82. doi: 10.1002/pd.3901. Epub 2012 Jun 13.

PMID:
22693141
31.

Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major.

Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A.

Haematologica. 2012 Jun;97(6):842-8. doi: 10.3324/haematol.2011.049957. Epub 2012 Jan 22.

32.

Risk factors for pulmonary hypertension in patients with β thalassemia intermedia.

Karimi M, Musallam KM, Cappellini MD, Daar S, El-Beshlawy A, Belhoul K, Saned MS, Temraz S, Koussa S, Taher AT.

Eur J Intern Med. 2011 Dec;22(6):607-10. doi: 10.1016/j.ejim.2011.05.013. Epub 2011 Jun 29.

PMID:
22075289
33.

Challenges of adherence and persistence with iron chelation therapy.

Porter JB, Evangeli M, El-Beshlawy A.

Int J Hematol. 2011 Nov;94(5):453-60. doi: 10.1007/s12185-011-0927-3. Epub 2011 Oct 13.

PMID:
21993873
34.

Importance of optimal dosing ≥ 30 mg/kg/d during deferasirox treatment: 2.7-yr follow-up from the ESCALATOR study in patients with β-thalassaemia.

Taher A, Elalfy MS, Al Zir K, Daar S, Al Jefri A, Habr D, Kriemler-Krahn U, El-Ali A, Roubert B, El-Beshlawy A.

Eur J Haematol. 2011 Oct;87(4):355-65. doi: 10.1111/j.1600-0609.2011.01662.x. Epub 2011 Jul 31.

35.

Achieving treatment goals of reducing or maintaining body iron burden with deferasirox in patients with β-thalassaemia: results from the ESCALATOR study.

Taher A, Elalfy MS, Al Zir K, Daar S, Al Jefri A, Habr D, Kriemler-Krahn U, Roubert B, El-Beshlawy A.

Eur J Haematol. 2011 Oct;87(4):349-54. doi: 10.1111/j.1600-0609.2011.01661.x. Epub 2011 Jul 26.

36.

The effect of reducing repetition time TR on the measurement of liver R2 for the purpose of measuring liver iron concentration.

Pavitt HL, Aydinok Y, El-Beshlawy A, Bayraktaroglu S, Ibrahim AS, Hamdy MM, Pang W, Sharples C, St Pierre TG.

Magn Reson Med. 2011 May;65(5):1346-51. doi: 10.1002/mrm.22712. Epub 2010 Nov 30.

37.

Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload.

Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A.

Haematologica. 2011 Jan;96(1):48-54. doi: 10.3324/haematol.2010.031468. Epub 2010 Nov 11.

38.

The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload.

ElAlfy MS, Sari TT, Lee CL, Tricta F, El-Beshlawy A.

J Pediatr Hematol Oncol. 2010 Nov;32(8):601-5. doi: 10.1097/MPH.0b013e3181ec0f13. Erratum in: J Pediatr Hematol Oncol. 2011 Oct;33(7):572. El Alfy, Moshen [corrected to ElAlfy, Moshen S].

PMID:
20921906
39.

ThalassoChip, an array mutation and single nucleotide polymorphism detection tool for the diagnosis of β-thalassaemia.

Shammas C, Papasavva T, Felekis X, Christophorou C, Roomere H, Synodinos JT, Kanavakis E, El-Khateeb M, Hamamy H, Mahmoud T, Shboul M, El Beshlawy A, Filon D, Hussein IR, Galanello R, Romeo G, Kleanthous M.

Clin Chem Lab Med. 2010 Dec;48(12):1713-8. doi: 10.1515/CCLM.2010.331. Epub 2010 Aug 13.

PMID:
20704537
40.

Splenectomy and thrombosis: the case of thalassemia intermedia.

Taher AT, Musallam KM, Karimi M, El-Beshlawy A, Belhoul K, Daar S, Saned M, Cesaretti C, Cappellini MD.

J Thromb Haemost. 2010 Oct;8(10):2152-8. doi: 10.1111/j.1538-7836.2010.03940.x.

41.

Age-related complications in treatment-naïve patients with thalassaemia intermedia.

Taher AT, Musallam KM, El-Beshlawy A, Karimi M, Daar S, Belhoul K, Saned MS, Graziadei G, Cappellini MD.

Br J Haematol. 2010 Aug;150(4):486-9. doi: 10.1111/j.1365-2141.2010.08220.x. Epub 2010 Apr 29. No abstract available.

PMID:
20456362
42.

Improved treatment satisfaction and convenience with deferasirox in iron-overloaded patients with beta-Thalassemia: Results from the ESCALATOR Trial.

Taher A, Al Jefri A, Elalfy MS, Al Zir K, Daar S, Rofail D, Baladi JF, Habr D, Kriemler-Krahn U, El-Beshlawy A.

Acta Haematol. 2010;123(4):220-5. doi: 10.1159/000313447. Epub 2010 Apr 27.

PMID:
20424435
43.

Neuronopathic Gaucher disease: demographic and clinical features of 131 patients enrolled in the International Collaborative Gaucher Group Neurological Outcomes Subregistry.

Tylki-Szymańska A, Vellodi A, El-Beshlawy A, Cole JA, Kolodny E.

J Inherit Metab Dis. 2010 Aug;33(4):339-46. doi: 10.1007/s10545-009-9009-6. Epub 2010 Jan 19.

PMID:
20084461
44.

Overview on practices in thalassemia intermedia management aiming for lowering complication rates across a region of endemicity: the OPTIMAL CARE study.

Taher AT, Musallam KM, Karimi M, El-Beshlawy A, Belhoul K, Daar S, Saned MS, El-Chafic AH, Fasulo MR, Cappellini MD.

Blood. 2010 Mar 11;115(10):1886-92. doi: 10.1182/blood-2009-09-243154. Epub 2009 Dec 23.

45.

Fetal globin induction in beta-thalassemia.

El-Beshlawy A, Hamdy M, El Ghamrawy M.

Hemoglobin. 2009;33 Suppl 1:S197-203. doi: 10.3109/03630260903351882. Review.

PMID:
20001626
46.

Prevention of hemoglobinopathies in Egypt.

El-Beshlawy A, Youssry I.

Hemoglobin. 2009;33 Suppl 1:S14-20. doi: 10.3109/03630260903346395.

PMID:
20001619
47.

Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia.

Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A.

Blood. 2010 Mar 25;115(12):2364-71. doi: 10.1182/blood-2009-04-217455. Epub 2009 Dec 8.

48.

Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias.

Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators.

Haematologica. 2010 Apr;95(4):557-66. doi: 10.3324/haematol.2009.014696. Epub 2009 Nov 30.

49.

Study of protein C, protein S, and antithrombin III in newborns with sepsis.

El Beshlawy A, Alaraby I, Abou Hussein H, Abou-Elew HH, Mohamed Abdel Kader MS.

Pediatr Crit Care Med. 2010 Jan;11(1):52-9. doi: 10.1097/PCC.0b013e3181c59032.

PMID:
19924026
50.

An electronic infrastructure for research and treatment of the thalassemias and other hemoglobinopathies: the Euro-mediterranean ITHANET project.

Lederer CW, Basak AN, Aydinok Y, Christou S, El-Beshlawy A, Eleftheriou A, Fattoum S, Felice AE, Fibach E, Galanello R, Gambari R, Gavrila L, Giordano PC, Grosveld F, Hassapopoulou H, Hladka E, Kanavakis E, Locatelli F, Old J, Patrinos GP, Romeo G, Taher A, Traeger-Synodinos J, Vassiliou P, Villegas A, Voskaridou E, Wajcman H, Zafeiropoulos A, Kleanthous M.

Hemoglobin. 2009;33(3):163-76. doi: 10.1080/03630260903089177.

PMID:
19657830

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