Correction of Severe Myelofibrosis, Impaired Platelet Functions and Abnormalities in a Patient with Gray Platelet Syndrome Successfully Treated by Stem Cell Transplantation
Gray platelet syndrome (GPS) is an inherited disorder. Patients harboring GPS have thrombocytopenia with large platelets lacking α-granules. A long-term complication is myelofibrosis with pancytopenia. Hematopoietic stem cell transplant (HSCT) could be a curative treatment. We report a male GPS patient with severe pancytopenia, splenomegaly and a secondary myelofibrosis needing red blood cells transfusion. He received an HSCT from a 10/10 matched HLA-unrelated donor after a myeloablative conditioning regimen. Transfusion independence occurred at day+21, with a documented neutrophil engraftment. At day+ 180, we added ruxolitinib to cyclosporine and steroids for a moderate chronic graft versus host disease (GVHD) and persistent splenomegaly. At day+240 GVHD was controlled and splenomegaly reduced. Complete donor chimesrism was documented in blood and marrow and platelets functions and morphology normalized. At day+ 720, the spleen size normalized and there was no evidence of marrow fibrosis on the biopsy. In GPS, HSCT may be a curative treatment in selected patients with pancytopenia and myelofibrosis.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2020 |
|---|---|
| Erschienen: |
2020 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:31 |
|---|---|
| Enthalten in: |
Platelets - 31(2020), 4 vom: 18. Mai, Seite 536-540 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Favier, Rémi [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
82S8X8XX8H |
|---|
| Anmerkungen: |
Date Completed 25.11.2020 Date Revised 04.12.2021 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1080/09537104.2019.1663809 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM301113351 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM301113351 | ||
| 003 | DE-627 | ||
| 005 | 20231225103945.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231225s2020 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1080/09537104.2019.1663809 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1003.xml |
| 035 | |a (DE-627)NLM301113351 | ||
| 035 | |a (NLM)31502501 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Favier, Rémi |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Correction of Severe Myelofibrosis, Impaired Platelet Functions and Abnormalities in a Patient with Gray Platelet Syndrome Successfully Treated by Stem Cell Transplantation |
| 264 | 1 | |c 2020 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 25.11.2020 | ||
| 500 | |a Date Revised 04.12.2021 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Gray platelet syndrome (GPS) is an inherited disorder. Patients harboring GPS have thrombocytopenia with large platelets lacking α-granules. A long-term complication is myelofibrosis with pancytopenia. Hematopoietic stem cell transplant (HSCT) could be a curative treatment. We report a male GPS patient with severe pancytopenia, splenomegaly and a secondary myelofibrosis needing red blood cells transfusion. He received an HSCT from a 10/10 matched HLA-unrelated donor after a myeloablative conditioning regimen. Transfusion independence occurred at day+21, with a documented neutrophil engraftment. At day+ 180, we added ruxolitinib to cyclosporine and steroids for a moderate chronic graft versus host disease (GVHD) and persistent splenomegaly. At day+240 GVHD was controlled and splenomegaly reduced. Complete donor chimesrism was documented in blood and marrow and platelets functions and morphology normalized. At day+ 720, the spleen size normalized and there was no evidence of marrow fibrosis on the biopsy. In GPS, HSCT may be a curative treatment in selected patients with pancytopenia and myelofibrosis | ||
| 650 | 4 | |a Case Reports | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Gray platelet syndrome | |
| 650 | 4 | |a inherited platelet disorder | |
| 650 | 4 | |a stem cell transplantation | |
| 650 | 7 | |a Nitriles |2 NLM | |
| 650 | 7 | |a Pyrazoles |2 NLM | |
| 650 | 7 | |a Pyrimidines |2 NLM | |
| 650 | 7 | |a ruxolitinib |2 NLM | |
| 650 | 7 | |a 82S8X8XX8H |2 NLM | |
| 650 | 7 | |a Cyclosporine |2 NLM | |
| 650 | 7 | |a 83HN0GTJ6D |2 NLM | |
| 700 | 1 | |a Roussel, Xavier |e verfasserin |4 aut | |
| 700 | 1 | |a Audia, Sylvain |e verfasserin |4 aut | |
| 700 | 1 | |a Bordet, Jean Claude |e verfasserin |4 aut | |
| 700 | 1 | |a De Maistre, Emmanuel |e verfasserin |4 aut | |
| 700 | 1 | |a Hirsch, Pierre |e verfasserin |4 aut | |
| 700 | 1 | |a Neuhart, Anne |e verfasserin |4 aut | |
| 700 | 1 | |a Bedgedjian, Isabelle |e verfasserin |4 aut | |
| 700 | 1 | |a Gkalea, Vasiliki |e verfasserin |4 aut | |
| 700 | 1 | |a Favier, Marie |e verfasserin |4 aut | |
| 700 | 1 | |a Daguindau, Etienne |e verfasserin |4 aut | |
| 700 | 1 | |a Nurden, Paquita |e verfasserin |4 aut | |
| 700 | 1 | |a Deconinck, Eric |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Platelets |d 1990 |g 31(2020), 4 vom: 18. Mai, Seite 536-540 |w (DE-627)NLM091628660 |x 1369-1635 |7 nnns |
| 773 | 1 | 8 | |g volume:31 |g year:2020 |g number:4 |g day:18 |g month:05 |g pages:536-540 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1080/09537104.2019.1663809 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 31 |j 2020 |e 4 |b 18 |c 05 |h 536-540 | ||