What is this summary about?Severe aplastic anemia (SAA) and very severe aplastic anemia (vSAA) are blood diseases of the bone marrow. If a suitable donor for bone marrow transplant as initial treatment is unavailable, standard immunosuppression is used. Standard immunosuppression treatment includes horse antithymocyte globulin (hATG) and cyclosporin A (CsA). This summary investigated the results of standard immunosuppression treatment (Group A) versus standard immunosuppression treatment with a medication called eltrombopag (Group B) in participants with SAA and vSAA. Eltrombopag is a medicine that improves the blood platelet level and is taken by mouth (orally).What were the results of the study?Compared to Group A, more participants in Group B showed increased blood cell level to a normal range without SAA or vSAA and faster treatment response. Side effects were similar in both groups even with the addition of eltrombopag for Group B. Participants in both groups reported feeling well after 6, 12 and 24 months. Differences in the participant-reported scores (overall health, physical, emotional, and social) between Group A and Group B were minimal.What do the results of the study mean? Immunosuppression treatment (hATG plus CsA) with eltrombopag benefited participants with SAA and vSAA and could be the new standard for SAA in persons who cannot undergo bone marrow transplant. At this time, eltrombopag is only approved in specific countries to treat the condition under study that is discussed in this summary. Clinical Trial Registration: NCT02099747 (RACE study)What do the results of the study mean?Immunosuppression treatment (hATG plus CsA) with eltrombopag benefited participants with SAA and vSAA and could be the new standard for SAA in persons who cannot undergo bone marrow transplant. At this time, eltrombopag is only approved in specific countries to treat the condition under study that is discussed in this summary. Clinical Trial Registration: NCT02099747 (RACE study)

de Latour, R.p., Kulasekararaj, A., Iacobelli, S., Griffin, M., Halkes, C.j., Dufour, C., et al. (2024). Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia. IMMUNOTHERAPY, 16(3) [10.2217/imt-2023-0200].

Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia

Iacobelli, Simona;
2024-02-01

Abstract

What is this summary about?Severe aplastic anemia (SAA) and very severe aplastic anemia (vSAA) are blood diseases of the bone marrow. If a suitable donor for bone marrow transplant as initial treatment is unavailable, standard immunosuppression is used. Standard immunosuppression treatment includes horse antithymocyte globulin (hATG) and cyclosporin A (CsA). This summary investigated the results of standard immunosuppression treatment (Group A) versus standard immunosuppression treatment with a medication called eltrombopag (Group B) in participants with SAA and vSAA. Eltrombopag is a medicine that improves the blood platelet level and is taken by mouth (orally).What were the results of the study?Compared to Group A, more participants in Group B showed increased blood cell level to a normal range without SAA or vSAA and faster treatment response. Side effects were similar in both groups even with the addition of eltrombopag for Group B. Participants in both groups reported feeling well after 6, 12 and 24 months. Differences in the participant-reported scores (overall health, physical, emotional, and social) between Group A and Group B were minimal.What do the results of the study mean? Immunosuppression treatment (hATG plus CsA) with eltrombopag benefited participants with SAA and vSAA and could be the new standard for SAA in persons who cannot undergo bone marrow transplant. At this time, eltrombopag is only approved in specific countries to treat the condition under study that is discussed in this summary. Clinical Trial Registration: NCT02099747 (RACE study)What do the results of the study mean?Immunosuppression treatment (hATG plus CsA) with eltrombopag benefited participants with SAA and vSAA and could be the new standard for SAA in persons who cannot undergo bone marrow transplant. At this time, eltrombopag is only approved in specific countries to treat the condition under study that is discussed in this summary. Clinical Trial Registration: NCT02099747 (RACE study)
feb-2024
Pubblicato
Rilevanza internazionale
Review
Sì, ma tipo non specificato
Settore MED/01
Settore MED/15
English
cyclosporin A; eltrombopag; horse antithymocyte globulin; lay summary; plain language summary; severe aplastic anemia; standard treatment
de Latour, R.p., Kulasekararaj, A., Iacobelli, S., Griffin, M., Halkes, C.j., Dufour, C., et al. (2024). Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia. IMMUNOTHERAPY, 16(3) [10.2217/imt-2023-0200].
de Latour, Rp; Kulasekararaj, A; Iacobelli, S; Griffin, M; Halkes, Cj; Dufour, C; Risitano, Am
Articolo su rivista
File in questo prodotto:
File Dimensione Formato  
Peffault de Latour et al 2023_Immunotherapy_RACE trial PLSP.pdf

accesso aperto

Tipologia: Versione Editoriale (PDF)
Licenza: Creative commons
Dimensione 1.6 MB
Formato Adobe PDF
1.6 MB Adobe PDF Visualizza/Apri

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2108/356044
Citazioni
  • ???jsp.display-item.citation.pmc??? 0
  • Scopus 0
  • ???jsp.display-item.citation.isi??? 0
social impact