Summary of findings and limitations of fetal hemoglobin inducer studies in patients with β-thalassemia
| Agent . | Main positive findings . | Limitations . |
|---|---|---|
| DNA methylation inhibitors | ||
| 5-azacytidine | Marked hematological responses achieved. | Few studies. |
| Small sample sizes. | ||
| Safety concerns. | ||
| Decitabine | Hematological responses achieved. | Few studies. |
| Favorable effects on red cell indices noted. | Small sample sizes. | |
| Treatment was well tolerated. | ||
| Hydroxyurea | Hematological responses achieved. | Heterogonous phenotypes studied together. |
| Favorable effects on red cell, hemolysis, and hypercoagulability indices noted. | Heterogeneous study end points evaluated together. | |
| Favorable effects on clinical morbidities noted. | Ideal dose and duration of therapy still controversial. | |
| Treatment was well tolerated. | Lack of efficacy on long-term therapy. | |
| Data on predictors of response still inconsistent. | ||
| Short-chain fatty acids | Hematological responses achieved. | Small sample sizes. |
| Favorable effects on red cell and hemolysis indices noted. | Lack of efficacy on long-term therapy. | |
| Treatment was well tolerated. | ||
| Erythropoietic-stimulating agents | Hematological responses achieved. | Few studies. |
| Favorable effects on combination with hydroxyurea noted. | Small sample sizes. | |
| Treatment was well tolerated. | High doses required. | |
| No additive effects with short-chain fatty acids. | ||
| Thalidomide and derivatives | Hematological responses achieved.Treatment was well tolerated. | Few studies.Small sample sizes. |
| Agent . | Main positive findings . | Limitations . |
|---|---|---|
| DNA methylation inhibitors | ||
| 5-azacytidine | Marked hematological responses achieved. | Few studies. |
| Small sample sizes. | ||
| Safety concerns. | ||
| Decitabine | Hematological responses achieved. | Few studies. |
| Favorable effects on red cell indices noted. | Small sample sizes. | |
| Treatment was well tolerated. | ||
| Hydroxyurea | Hematological responses achieved. | Heterogonous phenotypes studied together. |
| Favorable effects on red cell, hemolysis, and hypercoagulability indices noted. | Heterogeneous study end points evaluated together. | |
| Favorable effects on clinical morbidities noted. | Ideal dose and duration of therapy still controversial. | |
| Treatment was well tolerated. | Lack of efficacy on long-term therapy. | |
| Data on predictors of response still inconsistent. | ||
| Short-chain fatty acids | Hematological responses achieved. | Small sample sizes. |
| Favorable effects on red cell and hemolysis indices noted. | Lack of efficacy on long-term therapy. | |
| Treatment was well tolerated. | ||
| Erythropoietic-stimulating agents | Hematological responses achieved. | Few studies. |
| Favorable effects on combination with hydroxyurea noted. | Small sample sizes. | |
| Treatment was well tolerated. | High doses required. | |
| No additive effects with short-chain fatty acids. | ||
| Thalidomide and derivatives | Hematological responses achieved.Treatment was well tolerated. | Few studies.Small sample sizes. |