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Quoted phrase not found in phrase index: "Diamond-Blackfan anemia 21"
Page 1
Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes.
Bezzerri V, Api M, Allegri M, Fabrizzi B, Corey SJ, Cipolli M. Bezzerri V, et al. Int J Mol Sci. 2020 Jun 30;21(13):4672. doi: 10.3390/ijms21134672. Int J Mol Sci. 2020. PMID: 32630050 Free PMC article. Review.
The pathogenesis of IBMFS involves mutations in several genes which encode for proteins involved in DNA repair, telomere biology and ribosome biogenesis. The classical IBMFS include Shwachman-Diamond syndrome (SDS), Diamond-Blackfan anemia (DBA), Fanco …
The pathogenesis of IBMFS involves mutations in several genes which encode for proteins involved in DNA repair, telomere biology and ribosom …
Managing the Unusual Causes of Fetal Anemia.
Maisonneuve E, Ben M'Barek I, Leblanc T, Da Costa L, Friszer S, Pernot F, Thomas P, Castaigne V, Toly N'Dour C, Mailloux A, Cortey A, Jouannic JM. Maisonneuve E, et al. Fetal Diagn Ther. 2020;47(2):156-164. doi: 10.1159/000501554. Epub 2019 Sep 10. Fetal Diagn Ther. 2020. PMID: 31505487
Among those IUT, 23 (3.9%) were performed for a rare or unknown cause of anemia in 13 fetuses (5.1% of transfused fetuses). The median gestational age at diagnosis was 26 weeks of gestation (WG; range 21-33). ...There was 1 case with suspected type I CDA and 1 with …
Among those IUT, 23 (3.9%) were performed for a rare or unknown cause of anemia in 13 fetuses (5.1% of transfused fetuses). The media …
L-leucine improves anemia and growth in patients with transfusion-dependent Diamond-Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond-Blackfan Anemia Registry.
Vlachos A, Atsidaftos E, Lababidi ML, Muir E, Rogers ZR, Alhushki W, Bernstein J, Glader B, Gruner B, Hartung H, Knoll C, Loew T, Nalepa G, Narla A, Panigrahi AR, Sieff CA, Walkovich K, Farrar JE, Lipton JM. Vlachos A, et al. Pediatr Blood Cancer. 2020 Dec;67(12):e28748. doi: 10.1002/pbc.28748. Epub 2020 Oct 6. Pediatr Blood Cancer. 2020. PMID: 33025707 Free PMC article. Clinical Trial.
BACKGROUND: Diamond-Blackfan anemia (DBA) is an inherited bone marrow failure syndrome characterized by anemia, short stature, congenital anomalies, and cancer predisposition. ...In a small patient series and in animal models, there have been hematolog …
BACKGROUND: Diamond-Blackfan anemia (DBA) is an inherited bone marrow failure syndrome characterized by anemia, …
Successful treatment of Diamond-Blackfan anemia with interleukin 3.
Gillio AP, Faulkner LB, Alter BP, Reilly L, Klafter R, Heller G, Young DC, Lipton JM, Moore MA, O'Reilly RJ. Gillio AP, et al. Stem Cells. 1993 Jul;11 Suppl 2:123-30. doi: 10.1002/stem.5530110820. Stem Cells. 1993. PMID: 7691318 Clinical Trial.
This report describes the response of 18 Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin 3 (rhIL-3). rhIL-3 was administered s.c. once daily on an escalating dose schedule (0.5-10 micrograms/kg/day). The rhIL-3 dose was escalated every …
This report describes the response of 18 Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin 3 (rhI …
Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3.
Gillio AP, Faulkner LB, Alter BP, Reilly L, Klafter R, Heller G, Young DC, Lipton JM, Moore MA, O'Reilly RJ. Gillio AP, et al. Blood. 1993 Aug 1;82(3):744-51. Blood. 1993. PMID: 8338944 Free article. Clinical Trial.
This report describes the response of eighteen Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin-3 (rhIL-3). rhIL-3 was administered subcutaneously once daily on an escalating dose schedule (0.5 to 10 micrograms/kg/d). The rhIL-3 dose wa …
This report describes the response of eighteen Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin- …
High-dose intravenous methylprednisolone therapy for patients with Diamond-Blackfan anemia refractory to conventional doses of prednisone.
Bernini JC, Carrillo JM, Buchanan GR. Bernini JC, et al. J Pediatr. 1995 Oct;127(4):654-9. doi: 10.1016/s0022-3476(95)70134-6. J Pediatr. 1995. PMID: 7562296
OBJECTIVES: To assess the efficacy and toxicity of very high doses of glucocorticoids in patients with congenital pure red cell aplasia (Diamond-Blackfan anemia) who did not respond to standard doses of prednisone. STUDY DESIGNS: We prospectively treated eigh …
OBJECTIVES: To assess the efficacy and toxicity of very high doses of glucocorticoids in patients with congenital pure red cell aplasia ( …
Desferrioxamine ototoxicity in an adult transfusion-dependent population.
Chiodo AA, Alberti PW, Sher GD, Francombe WH, Tyler B. Chiodo AA, et al. J Otolaryngol. 1997 Apr;26(2):116-22. J Otolaryngol. 1997. PMID: 9106087
OBJECTIVE: The purpose of this study was to identify the incidence of hearing loss in a population of 75 adult (19-68 years old) transfusion-dependent patients with thalassemia major, sickle cell disease, Diamond-Blackfan anemia, and various other hematologic …
OBJECTIVE: The purpose of this study was to identify the incidence of hearing loss in a population of 75 adult (19-68 years old) transfusion …
Is there a role for interleukin-3 in Diamond-Blackfan anaemia? Results of a European multicentre study.
Ball SE, Tchernia G, Wranne L, Bastion Y, Bekassy NA, Bordigoni P, Debré M, Elinder G, Kamps WA, Lanning M, et al. Ball SE, et al. Br J Haematol. 1995 Oct;91(2):313-8. doi: 10.1111/j.1365-2141.1995.tb05295.x. Br J Haematol. 1995. PMID: 8547067 Clinical Trial.
Forty patients (nine adults aged 20-54; 31 children aged 1-17) with Diamond-Blackfan anaemia (DBA) were treated with recombinant human interleukin-3 (IL-3) in a European multicentre compassionate-need study. IL-3 was given as a daily subcutaneous injection at …
Forty patients (nine adults aged 20-54; 31 children aged 1-17) with Diamond-Blackfan anaemia (DBA) were treated with re …
Reduced-intensity stem cell transplantation in children and adolescents: the Mexican experience.
Gómez-Almaguer D, Ruiz-Argüelles GJ, Tarín-Arzaga Ldel C, González-Llano O, Jaime-Pérez JC, López-Martínez B, Cantú-Rodríguez OG, Herrera-Garza JL. Gómez-Almaguer D, et al. Biol Blood Marrow Transplant. 2003 Mar;9(3):157-61. doi: 10.1053/bbmt.2003.50004. Biol Blood Marrow Transplant. 2003. PMID: 12652465 Free article. Clinical Trial.
A group of 21 consecutive patients aged 4-20 (median 13) years was prospectively allografted using a reduced intensity preparative regimen. The group included both malignant (acute lymphoblastic leukemia, acute myelogenous leukemia, and chronic myelogenous leukemia) and no …
A group of 21 consecutive patients aged 4-20 (median 13) years was prospectively allografted using a reduced intensity preparative re …
Oral iron chelation therapy with deferiprone. Monitoring of biochemical, drug and iron excretion changes.
Kontoghiorghes GJ, Bartlett AN, Sheppard L, Barr J, Nortey P. Kontoghiorghes GJ, et al. Arzneimittelforschung. 1995 Jan;45(1):65-9. Arzneimittelforschung. 1995. PMID: 7893273 Clinical Trial.
The oral iron chelator 1,2-dimethyl-3-hydroxypyrid-4-one (L1, deferiprone, CAS 30652-11-0) has been given daily for 3-11 months to 6 transfusion dependent iron loaded patients (myelodysplasia (MDS) 2, Diamond-Blackfan anaemia 1, thalassaemia intermedia 1, tha …
The oral iron chelator 1,2-dimethyl-3-hydroxypyrid-4-one (L1, deferiprone, CAS 30652-11-0) has been given daily for 3-11 months to 6 transfu …
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