TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT
Systemic mastocytosis (SM) is a rare disease with a range of clinical presentations, and the vast majority of patients have a KIT D816V mutation that results in gain of function. The multikinase/KIT inhibitor midostaurin inhibits the D816V mutant, and has a well-established role in treatment of adv...
Saved in:
| Main Authors: | , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
PAGEPress Publications
2022-10-01
|
| Series: | Mediterranean Journal of Hematology and Infectious Diseases |
| Subjects: | |
| Online Access: | http://mjhid.org/index.php/mjhid/article/view/5042 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1850059517088235520 |
|---|---|
| author | Cristina Papayannidis Vincenzo Federico Luana Fianchi Patrizia Pregno Novella Pugliese Alessandra Romano Federica Irene Grifoni |
| author_facet | Cristina Papayannidis Vincenzo Federico Luana Fianchi Patrizia Pregno Novella Pugliese Alessandra Romano Federica Irene Grifoni |
| author_sort | Cristina Papayannidis |
| collection | DOAJ |
| description |
Systemic mastocytosis (SM) is a rare disease with a range of clinical presentations, and the vast majority of patients have a KIT D816V mutation that results in gain of function. The multikinase/KIT inhibitor midostaurin inhibits the D816V mutant, and has a well-established role in treatment of advanced SM. Even if considered the standard of therapy, some open questions remain on how to optimize the management of midostaurin in daily practice. The current review presents the opinions of a group of experts who met to discuss routine practice with the use of midostaurin in patients with advanced SM. The efficacy and safety of midostaurin in Phase 2 trials are overviewed, followed by practical guidance for optimal management of therapy and adverse events during therapy with midostaurin. Specific guidance is given for initiating therapy and evaluating response with midostaurin in terms of general assessment and laboratory, instrumental, pathological, and molecular exams. Special consideration is given to dose interruption, reduction, and discontinuation of therapy as well as adverse event management and supportive therapy. Patients should be informed about possible side effects and receive not only practical advice to avoid or limit them, but also antiemetic prophylaxis so that therapy with midostaurin can continue as long as clinical benefit is observed or until unacceptable toxicity occurs. Lastly, considerations on the use of midostaurin during the ongoing Covid-19 pandemic are made. The overall scope is to provide guidance that can be useful in daily practice for clinicians using midostaurin to treat patients with advanced SM.
|
| format | Article |
| id | doaj-art-728dfa3deedd466b81bfbaf974bbaaa5 |
| institution | DOAJ |
| issn | 2035-3006 |
| language | English |
| publishDate | 2022-10-01 |
| publisher | PAGEPress Publications |
| record_format | Article |
| series | Mediterranean Journal of Hematology and Infectious Diseases |
| spelling | doaj-art-728dfa3deedd466b81bfbaf974bbaaa52025-08-20T02:50:52ZengPAGEPress PublicationsMediterranean Journal of Hematology and Infectious Diseases2035-30062022-10-0114110.4084/MJHID.2022.073TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENTCristina Papayannidis0Vincenzo Federico1Luana Fianchi2Patrizia Pregno3Novella Pugliese4Alessandra Romano5Federica Irene Grifoni6Cristina Papayannidis, Department of Experimental, Diagnostic and Specialty Medicine, Bologna University, via Massarenti 9, 40138 Bologna, Italy. Tel.: +39.051.636.3973 - Fax: +39.051.636.4037. E-mail: cristina.papayannidis@unibo.itHaematology and Stem Cell Transplant Unit, Presidio Ospedaliero "Vito Fazzi", Lecce, ItalyDepartment of Radiological Sciences, Radiotherapy, and Hematology, Fondazione Policlinico Universitario A. Gemelli, IRCCS, Rome, ItalyHematology Division, Oncology and Hematology Department, AOU Città della Salute e della Scienza di Torino,Turin, ItalyDepartment of Clinical Medicine and Surgery, Hematology Unit, Federico II University Medical School, Naples, ItalyDivision of Hematology, Azienda Ospedaliero Universitaria Policlinico-Vittorio Emanuele Catania, Catania, ItalyHematology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan Italy Systemic mastocytosis (SM) is a rare disease with a range of clinical presentations, and the vast majority of patients have a KIT D816V mutation that results in gain of function. The multikinase/KIT inhibitor midostaurin inhibits the D816V mutant, and has a well-established role in treatment of advanced SM. Even if considered the standard of therapy, some open questions remain on how to optimize the management of midostaurin in daily practice. The current review presents the opinions of a group of experts who met to discuss routine practice with the use of midostaurin in patients with advanced SM. The efficacy and safety of midostaurin in Phase 2 trials are overviewed, followed by practical guidance for optimal management of therapy and adverse events during therapy with midostaurin. Specific guidance is given for initiating therapy and evaluating response with midostaurin in terms of general assessment and laboratory, instrumental, pathological, and molecular exams. Special consideration is given to dose interruption, reduction, and discontinuation of therapy as well as adverse event management and supportive therapy. Patients should be informed about possible side effects and receive not only practical advice to avoid or limit them, but also antiemetic prophylaxis so that therapy with midostaurin can continue as long as clinical benefit is observed or until unacceptable toxicity occurs. Lastly, considerations on the use of midostaurin during the ongoing Covid-19 pandemic are made. The overall scope is to provide guidance that can be useful in daily practice for clinicians using midostaurin to treat patients with advanced SM. http://mjhid.org/index.php/mjhid/article/view/5042systemic mastocytosismidostauringuidancemanagement |
| spellingShingle | Cristina Papayannidis Vincenzo Federico Luana Fianchi Patrizia Pregno Novella Pugliese Alessandra Romano Federica Irene Grifoni TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT Mediterranean Journal of Hematology and Infectious Diseases systemic mastocytosis midostaurin guidance management |
| title | TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT |
| title_full | TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT |
| title_fullStr | TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT |
| title_full_unstemmed | TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT |
| title_short | TREATMENT OF ADVANCED SYSTEMIC MASTOCYTOSIS WITH MIDOSTAURIN: PRACTICAL GUIDANCE FOR OPTIMAL THERAPY AND MANAGEMENT |
| title_sort | treatment of advanced systemic mastocytosis with midostaurin practical guidance for optimal therapy and management |
| topic | systemic mastocytosis midostaurin guidance management |
| url | http://mjhid.org/index.php/mjhid/article/view/5042 |
| work_keys_str_mv | AT cristinapapayannidis treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement AT vincenzofederico treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement AT luanafianchi treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement AT patriziapregno treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement AT novellapugliese treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement AT alessandraromano treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement AT federicairenegrifoni treatmentofadvancedsystemicmastocytosiswithmidostaurinpracticalguidanceforoptimaltherapyandmanagement |