Table 5.
Class | Molecule | In vitro study | Clinical trials in MF | Selected references | Trial planned/ongoing |
---|---|---|---|---|---|
Aim: to reduce unwanted side effects (anemia, thrombocytopenia) yet maintaining clinical benefits | |||||
Erythropoietin-stimulating agents | Erythropoietin or biosimilar | – | √ | Cervantes et al. [2006] | – |
TPO mimetics | Romiplostin | – | Kuter [2010] | – | |
Androgenic steroids | Danazol | – | √ | Cervantes et al. [2005] | Planned |
Immunomodulator | Pomalidomide | – | √ | Tefferi et al. [2009] | Planned |
Aim: disease modulation, i.e. to increase the benefits seen with JAK2 inhibitors and bring additional benefits | |||||
Immunomodulators | Pomalidomide | – | √ | Tefferi et al. [2009] | Planned |
Lenalidomide | – | √ | Mesa et al. [2010] | Ongoing | |
mTOR inhibitors | Everolimus | √ | √ | Guglielmelli et al. [2011a] | Planned |
Hypomethylating agents | Azacitidine | √ | √ | Mesa et al. [2009] | – |
Decitabine | √ | √ | Danilov et al. [2009] | – | |
Histone deacethylase inhibitors | Givinostat | √ | √ | Rambaldi et al. [2010] | – |
Panobinostat | √ | √ | Deangelo et al. [2010] | Ongoing | |
BCL-2 inhibitors | Obatoclax mesylate | √ | √ | Parikh et al. [2010] | – |
BCL-XL inhibitors | ABT-737 | √ | Lu et al. [2010] | – | |
HSP90 inhibitors | PU-H71 | √ | Marubayashi et al. [2010] | – | |
Pegylated interferon α – 2a | PEGASYS | √ | Kiladjian et al. [2008] | Planned | |
Aim: to facilitate stem cell transplantation | |||||
Stem cell transplant | NA | √ | Ongoing |
Information about ‘planned trials’ is to the best of authors’ current knowledge.
BCL, B-cell lymphoma; HSP, heat shock protein; JAK, Janus kinase; MF, myelofibrosis; mTOR, mammalian target of rapamycin; NA, not available; TPO, thrombopoietin.