Ongoing clinical trials in HLH
| Research group . | Clinical trial identifier . | Drug or treatment . | Region . | Years . | Overall survival, % . | Reference . | Target . | Status . |
|---|---|---|---|---|---|---|---|---|
| Beijing Friendship Hospital | NCT02631109 | Pegaspargase, doxorubicin, etoposide, methylprednisolone | Beijing, China | 2015-present | 46 | 114 | EBV-HLH | Recruiting |
| Children's Hospital of Philadelphia | NCT02007239 | Tocilizumab | Philadelphia, PA | 2013-present | NA | NA | IL-6 receptor | Recruiting |
| Children's Hospital Medical Center Cincinnati | NCT01104025 | ATG, etoposide, dexamethasone/hydrocortisone | Cincinnati, OH | 2010-2016 | NA | NA | T cells, proliferation | Complete, data pending |
| Karolinska University Hospital | NCT00426101 | Dexamethasone, etoposide, cyclosporine A | Multiple | 2007-present | 55 | 6 | Proliferation | Ongoing |
| Beijing Friendship Hospital | NCT02862054 | Splenectomy | Beijing, China | 2016-present | 25 | 115 | Splenectomy | Ongoing |
| Assistance Publique - Hôpitaux de Paris | NCT02472054 | Alemtuzumab, prednisone/methylprednisolone, cyclosporine A | Paris, France | 2015-present | NA | NA | CD-52, proliferation | Ongoing |
| University of Michigan Cancer Center | NCT02400463 | Ruxolitinib | Ann Arbor, MI | 2015-present | NA | NA | Jak1/2 | Ongoing |
| NovImmune SA | NCT01818492 | NI-0501 (anti-IFN-γ) | Multiple | 2013-present | NA | NA | IFN-γ | Ongoing |
| Peking University People's Hospital | NCT02569463 | IL-2 | Beijing, China | 2014-present | NA | NA | BM suppression | Ongoing |
| Baylor College of Medicine | NCT01494103 | T cells with iCaspase-9 | Houston, TX | 2011-present | NA | 116 | BM suppression | Ongoing |
| Masonic Cancer Center | NCT00176865 | HSCT | Minneapolis, MN | 2002-present | NA | NA | BM suppression | Ongoing |
| Masonic Cancer Center | NCT00176826 | HSCT | Minneapolis, MN | 2000-present | NA | NA | BM suppression | Ongoing |
| Washington University School of Medicine | NCT01821781 | HSCT | St. Louis, MO | 2013-present | NA | NA | BM suppression | Ongoing |
| Masonic Cancer Center | NCT01652092 | HSCT | Minneapolis, MN | 2012-present | NA | NA | BM suppression | Ongoing |
| Pusan National University Medical School | — | CHOP | Busan, South Korea | 1999-2004 | 44 | 117 | Proliferation | Complete |
| Research group . | Clinical trial identifier . | Drug or treatment . | Region . | Years . | Overall survival, % . | Reference . | Target . | Status . |
|---|---|---|---|---|---|---|---|---|
| Beijing Friendship Hospital | NCT02631109 | Pegaspargase, doxorubicin, etoposide, methylprednisolone | Beijing, China | 2015-present | 46 | 114 | EBV-HLH | Recruiting |
| Children's Hospital of Philadelphia | NCT02007239 | Tocilizumab | Philadelphia, PA | 2013-present | NA | NA | IL-6 receptor | Recruiting |
| Children's Hospital Medical Center Cincinnati | NCT01104025 | ATG, etoposide, dexamethasone/hydrocortisone | Cincinnati, OH | 2010-2016 | NA | NA | T cells, proliferation | Complete, data pending |
| Karolinska University Hospital | NCT00426101 | Dexamethasone, etoposide, cyclosporine A | Multiple | 2007-present | 55 | 6 | Proliferation | Ongoing |
| Beijing Friendship Hospital | NCT02862054 | Splenectomy | Beijing, China | 2016-present | 25 | 115 | Splenectomy | Ongoing |
| Assistance Publique - Hôpitaux de Paris | NCT02472054 | Alemtuzumab, prednisone/methylprednisolone, cyclosporine A | Paris, France | 2015-present | NA | NA | CD-52, proliferation | Ongoing |
| University of Michigan Cancer Center | NCT02400463 | Ruxolitinib | Ann Arbor, MI | 2015-present | NA | NA | Jak1/2 | Ongoing |
| NovImmune SA | NCT01818492 | NI-0501 (anti-IFN-γ) | Multiple | 2013-present | NA | NA | IFN-γ | Ongoing |
| Peking University People's Hospital | NCT02569463 | IL-2 | Beijing, China | 2014-present | NA | NA | BM suppression | Ongoing |
| Baylor College of Medicine | NCT01494103 | T cells with iCaspase-9 | Houston, TX | 2011-present | NA | 116 | BM suppression | Ongoing |
| Masonic Cancer Center | NCT00176865 | HSCT | Minneapolis, MN | 2002-present | NA | NA | BM suppression | Ongoing |
| Masonic Cancer Center | NCT00176826 | HSCT | Minneapolis, MN | 2000-present | NA | NA | BM suppression | Ongoing |
| Washington University School of Medicine | NCT01821781 | HSCT | St. Louis, MO | 2013-present | NA | NA | BM suppression | Ongoing |
| Masonic Cancer Center | NCT01652092 | HSCT | Minneapolis, MN | 2012-present | NA | NA | BM suppression | Ongoing |
| Pusan National University Medical School | — | CHOP | Busan, South Korea | 1999-2004 | 44 | 117 | Proliferation | Complete |
Current trials for treatment of HLH are limited in regard to established data. The HLH protocol remains the most effective therapy option to date (55%), but new innovations, especially in immunotherapy and kinase inhibitors, present new opportunities for increased efficacy. Some trials have looked at augmenting the HLH-2004 protocol with ATG or pegaspargase, but results have been limited by comparison with the HLH-2004 trial.
NA, not available.