Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia
Inherited thrombocytopenias represents a heterogenous group of diseases characterized by a congenital reduction in the platelet count that could lead to a bleeding tendency. MYH9-related disorders are characterized by large platelets and congenital thrombocytopenia. Thrombopoietin-receptor agonists:...
| Main Authors: | , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2022-11-01
|
| Series: | Children |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2227-9067/9/12/1839 |
| _version_ | 1827641356121538560 |
|---|---|
| author | Giuseppe Lassandro Francesco Carriero Domenico Noviello Valentina Palladino Giovanni Carlo Del Vecchio Maria Felicia Faienza Paola Giordano |
| author_facet | Giuseppe Lassandro Francesco Carriero Domenico Noviello Valentina Palladino Giovanni Carlo Del Vecchio Maria Felicia Faienza Paola Giordano |
| author_sort | Giuseppe Lassandro |
| collection | DOAJ |
| description | Inherited thrombocytopenias represents a heterogenous group of diseases characterized by a congenital reduction in the platelet count that could lead to a bleeding tendency. MYH9-related disorders are characterized by large platelets and congenital thrombocytopenia. Thrombopoietin-receptor agonists: eltrombopag and romiplostim are currently approved in many countries for the treatment of different forms of acquired thrombocytopenia, such as immune thrombocytopenia. We report, instead, the successful use of eltrombopag to treat inherited thrombocytopenia in a patient with an MHY9-related disease. This is the first report of a chronic use of eltrombopag to elevate platelets in MYH9-related disorders without side effects. |
| first_indexed | 2024-03-09T17:11:51Z |
| format | Article |
| id | doaj.art-dd8d45d00d274173a418e588a55553c1 |
| institution | Directory Open Access Journal |
| issn | 2227-9067 |
| language | English |
| last_indexed | 2024-03-09T17:11:51Z |
| publishDate | 2022-11-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Children |
| spelling | doaj.art-dd8d45d00d274173a418e588a55553c12023-11-24T14:00:38ZengMDPI AGChildren2227-90672022-11-01912183910.3390/children9121839Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited ThrombocytopeniaGiuseppe Lassandro0Francesco Carriero1Domenico Noviello2Valentina Palladino3Giovanni Carlo Del Vecchio4Maria Felicia Faienza5Paola Giordano6Azienda Ospedaliero Universitaria Consorziale Policlinico-Giovanni XXIII, Pediatria Universitaria “B. Trambusti”, 70126 Bari, ItalyInterdisciplinay Medicine Department, Pediatric Section, University of Bari “Aldo Moro”, 70126 Bari, ItalyInterdisciplinay Medicine Department, Pediatric Section, University of Bari “Aldo Moro”, 70126 Bari, ItalyAzienda Ospedaliero Universitaria Consorziale Policlinico-Giovanni XXIII, Pediatria Universitaria “B. Trambusti”, 70126 Bari, ItalyAzienda Ospedaliero Universitaria Consorziale Policlinico-Giovanni XXIII, Pediatria Universitaria “B. Trambusti”, 70126 Bari, ItalyAzienda Ospedaliero Universitaria Consorziale Policlinico-Giovanni XXIII, Pediatria Universitaria “B. Trambusti”, 70126 Bari, ItalyAzienda Ospedaliero Universitaria Consorziale Policlinico-Giovanni XXIII, Pediatria Universitaria “B. Trambusti”, 70126 Bari, ItalyInherited thrombocytopenias represents a heterogenous group of diseases characterized by a congenital reduction in the platelet count that could lead to a bleeding tendency. MYH9-related disorders are characterized by large platelets and congenital thrombocytopenia. Thrombopoietin-receptor agonists: eltrombopag and romiplostim are currently approved in many countries for the treatment of different forms of acquired thrombocytopenia, such as immune thrombocytopenia. We report, instead, the successful use of eltrombopag to treat inherited thrombocytopenia in a patient with an MHY9-related disease. This is the first report of a chronic use of eltrombopag to elevate platelets in MYH9-related disorders without side effects.https://www.mdpi.com/2227-9067/9/12/1839eltrombopagchildrenMYH9 |
| spellingShingle | Giuseppe Lassandro Francesco Carriero Domenico Noviello Valentina Palladino Giovanni Carlo Del Vecchio Maria Felicia Faienza Paola Giordano Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia Children eltrombopag children MYH9 |
| title | Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia |
| title_full | Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia |
| title_fullStr | Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia |
| title_full_unstemmed | Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia |
| title_short | Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia |
| title_sort | successful eltrombopag therapy in a child with myh9 related inherited thrombocytopenia |
| topic | eltrombopag children MYH9 |
| url | https://www.mdpi.com/2227-9067/9/12/1839 |
| work_keys_str_mv | AT giuseppelassandro successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia AT francescocarriero successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia AT domeniconoviello successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia AT valentinapalladino successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia AT giovannicarlodelvecchio successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia AT mariafeliciafaienza successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia AT paolagiordano successfuleltrombopagtherapyinachildwithmyh9relatedinheritedthrombocytopenia |