Primary Myelofibrosis
Watchlist
Retrieved
2022-04-26
Source
Trials
—
Genes
JAK2,
MPL,
CALR,
TET2,
SH2B3,
EZH2,
GATA1,
MYB,
NCOR2,
TP53,
MPIG6B,
NBEAL2,
THPO,
GFI1B,
CD34,
ABL1,
TGFB1,
TBXAS1,
SRC,
SRSF2,
JAK1,
SOAT1,
STAT5A,
BCR,
ASXL1,
STAT5B,
IDH1,
IDH2,
CXCR4,
CD177
JAK2,
MPL,
CALR,
TET2,
SH2B3,
EZH2,
GATA1,
MYB,
NCOR2,
TP53,
MPIG6B,
NBEAL2,
THPO,
GFI1B,
CD34,
ABL1,
TGFB1,
TBXAS1,
SRC,
SRSF2,
JAK1,
SOAT1,
STAT5A,
BCR,
ASXL1,
STAT5B,
IDH1,
IDH2,
CXCR4,
CD177,
PRB1,
EPO,
CXCL12,
HMGA2,
FN1,
TNFRSF11B,
LINC01152,
KIT,
STAT3,
CXCL8,
PDGFRA,
MVD,
FGF2,
DERL1,
AKT1,
IFNA1,
ATN1,
IFNA13,
SF3B1,
VEGFA,
VPS45,
PDGFRB,
MMRN1,
TNF,
TAC1,
FLT3,
CSF3,
FGFR1,
AURKA,
RUNX1,
IFNA2,
GLI1,
IRAK1,
LOX,
WNT1,
IL6,
WT1,
IL1B,
U2AF1,
LGALS3,
HDAC9,
FXYD5,
MIR34A,
LEF1,
NRAS,
SETBP1,
PIK3CA,
PIK3CB,
PIK3CD,
INTS2,
NOG,
MAPK3,
IL1A,
RARB,
SCT,
SOCS3,
PIK3CG,
H3P10,
NR3C1,
CDKN2A,
CD47,
CSF3R,
CCN2,
CTNNB1,
DNMT3A,
ETV6,
FGF3,
FKBP5,
FLT1,
FKBP4,
IFNG,
ALB,
MIR155,
ABI1,
HSPB3,
CALB2,
MIR4319,
MYOM2,
SLC25A20,
SOCS6,
CA2,
BTF3P11,
HDAC6,
IL18R1,
BRCA2,
HMGXB4,
EBI3,
GDF11,
STUB1,
MRPL28,
SUB1,
FGFR1OP,
PROM1,
CD164,
DLK1,
ZMYM2,
CRISP2,
TRPC4,
TYK2,
TYROBP,
VDR,
CD63,
H3P9,
CD14,
CD9,
MIR143,
SHOC2,
CBL,
CLLS2,
RN7SL263P,
BCL2L2-PABPN1,
CAT,
CNTNAP1,
ENC1,
NRM,
BNIP3,
MIR543,
BMP7,
INTS3,
XIAP,
BORA,
HDAC11,
AMCN,
ALPI,
FIP1L1,
PRSS27,
MAGT1,
SPZ1,
BMF,
FREM1,
ZFPM1,
ALPP,
PAOX,
PRSS55,
ZBTB8OS,
MIR127,
MIR140,
CIP2A,
STIM2,
STS,
INTS1,
HMGXB3,
BMP6,
BMP1,
MIR494,
CDKN2B,
MIR146B,
MIR382,
BID,
SGSM3,
KIDINS220,
NXT1,
PYCARD,
TBK1,
BCL2L2,
BCL2,
BAX,
AGGF1,
BAK1,
TPO,
IGF2,
TNFRSF1B,
LGALS1,
LIG3,
LIG4,
MCAM,
MMP8,
MMP13,
MMP14,
GLS,
GABPA,
G6PD,
MYC,
GADD45B,
CEACAM6,
NFE2,
NFE2L2,
NOS2,
NOS3,
NPM1,
FZD2,
NR4A2,
HIF1A,
LEPR,
PAEP,
LEP,
IFI27,
IL1RN,
IL2RA,
IL3,
IAPP,
HSPB2,
CXCR1,
CXCR2,
HSPB1,
ITGA9,
ITGB2,
HPGD,
HP,
JARID2,
JUN,
HMGA1,
KRT7,
LDHA,
LDHB,
MTOR,
PDGFB,
CEACAM5,
SNRPF,
SOD2,
SPARC,
SPP1,
ACE,
STAT1,
CUX1,
CSF1R,
CRP,
PRDX2,
CHIT1,
TGFBR1,
TGFBR2,
THBD,
THBS1,
THBS2,
CCN1,
THRB,
THY1,
TLR4,
DNMT1,
SLCO2A1,
FCGR2A,
SELP,
ACTB,
PLK1,
PPARG,
PRG2,
MAPK1,
EPOR,
MAPK8,
PTH,
PTHLH,
PTK2,
ENG,
RARS1,
RET,
RPS14,
S100A8,
S100A9,
S100A12,
ELN,
EDA,
CHI3L1
Drugs
11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene,
2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus
11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene,
2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate,
Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus,
Fedratinib
(
INREBIC
),
H-Tyrosine-Glycine-Phenylalanine-Glycine-Glycine-OH,
Imetelstat,
Luspatercept
(
REBLOZYL
),
Momelotinib,
N-[(2S)-5-{[(1R, 2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosylate salt,
N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate,
Navitoclax,
Plitidepsin,
Pomalidomide
(
IMNOVID (previously POMALIDOMIDE CELGENE),
POMALYST
),
Recombinant human pentraxin-2,
Ruxolitinib
(
JAKAFI,
JAKAVI
),
Sotatercept,
haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate
Registered!
Primary myelofibrosis is a condition characterized by the buildup of scar tissue (fibrosis) in the spongy tissue tissue inside the bone (bone marrow), the tissue that contains the stem cells that will produce blood cells. Because of the fibrosis, the bone marrow is unable to make enough normal blood cells. In myelofibrosis, the bone marrow is replaced by fibrous (scar) tissue. When the bone marrow is scarred, it cannot make enough blood cells. This leads to anemia, weakness, fatigue, and often, swelling of the liver and spleen. The disorder occurs when blood stem cells develop somatic mutations in the JAK2, MPL, CALR, and TET2 genes. Other genes may also be involved. The disorder is generally not inherited because this type of mutation does not affect the reproductive cells (sperm and egg) only certain cells of the body (somatic). Although myelofibrosis can occur at any age, it typically develops after the age of 50 years. In most cases, myelofibrosis gets progressively worse. Treatment is aimed at relieving signs and symptoms and may include medications, blood transfusions, chemotherapy, radiation therapy, and surgery. Bone marrow or stem cell transplant may improve symptoms, and may cure the disease.