Clinical response to eltrombopag therapy
| Subject number . | Platelet count (109/L) . | Median mean platelet volume . | Dosing (mg/d) . | Bleeding (1 = bleeding before and during tx; 2 = no bleeding before or during tx; 3 = bleeding reduced on tx) . | Change in platelet transfusions while on eltrombopag (0 = no transfusions prior to or during tx) . | Change in WAS/ XLT tx while on eltrombopag (0 = not on other medication prior to or during tx) . | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline count . | Highest count on epag (wk) . | Still on epag? (Y/N) . | Count at most recent follow- up . | Pre-epag . | On-epag . | Starting dose . | Maximum dose . | ||||
| Responders | |||||||||||
| 6 | 19 | 115 wk 64 | N | 11 20 wks off epag | 7.0 | 6.9 | 50 0.62 mg/kg | 75 0.88 mg/kg | 3 Frequent episodes of epistaxis, at least once per month lasting ≥30 min. On epag, fewer episodes that rarely lasted 10 min | 0 | 0 |
| 7 | 16 | 154 wk 152 | Y | 154 wk 152 | 5.7 | 6.3 | 35 1.9 mg/kg | 75 2.8 mg/kg | 3 Frequent bruising in addition to ICH and very occasional epistaxis that lasted ≥15 min. On epag, no bleeding with increased dose to 75 mg at month 18 | No platelet transfusions required while on epag (had required them prior to therapy for ICH) | Discontinued daily amicar while on epag |
| 8* | 44 | 98 wks 82 and 111 | Y | 47 wk 209 | 5.4 | 6.2 | 9 0.7 mg/kg | 75 3.3 mg/kg | 3 Substantial bruising, oral bleeding, and epistaxis. On epag, fewer episodes of epistaxis and decreased bruising | Less frequent platelet transfusions required (only 1 not related to surgical procedures) | No change in WAS/XLT tx |
| 9 | 20 | 134 wk 126 | Y | 134 wk 126 | 9 | 7.1 | 20 1.6 mg/kg | 60 3.8 mg/kg | 3 Frequent bruising, constant petechiae, and one major bleed when hitting lip. On epag, no bruising or bleeding. Pt is more energetic while on epag | 0 | 0 IVIG ×1 while on epag when pt had a viral infection |
| 12† | 14 | 283 wk 22 | N | 128 24 wks off epag | 6.2 | 5.8 | 25 1.6 mg/kg | 75 5 mg/kg | 2 Before prednisone and epag, bruising and other bleeding. No bleeding on prednisone alone. With addition of epag, no bleeding, including when prednisone was subsequently discontinued | 0 | Discontinued daily prednisone (0.2 mg/kg) while on epag |
| Nonresponders | |||||||||||
| 10 | 9 | 166 wk 6 (after platelet trans-fusion) | N | Lost to follow-up | 7.2 | 8.2 | 50 1.3 mg/kg | 75 2.1 mg/kg | 1 | Frequent platelet transfusions required before and during epag therapy | 0 |
| 11 | 15 | 37 wk 187 | Y | 17 wk 198 | 7.7 | 8.3 | 25 1.1 mg/kg | 75 2.8 mg/kg | 3 Bruising and other minor bleeding. On epag, no bleeding. Pt is more energetic on epag. | Does not require platelet transfusions (had required weekly platelet transfusions prior to epag) | 0 |
| 13 | 13 | 55 wk 2 | N | 30 58 wks off epag, while on romiplostim | 9.5 | 8.2 | 50 weight n/a | 75 weight n/a | 3 On epag, slightly less skin and mucous membrane bleeding | 0 | 0 |
| Subject number . | Platelet count (109/L) . | Median mean platelet volume . | Dosing (mg/d) . | Bleeding (1 = bleeding before and during tx; 2 = no bleeding before or during tx; 3 = bleeding reduced on tx) . | Change in platelet transfusions while on eltrombopag (0 = no transfusions prior to or during tx) . | Change in WAS/ XLT tx while on eltrombopag (0 = not on other medication prior to or during tx) . | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline count . | Highest count on epag (wk) . | Still on epag? (Y/N) . | Count at most recent follow- up . | Pre-epag . | On-epag . | Starting dose . | Maximum dose . | ||||
| Responders | |||||||||||
| 6 | 19 | 115 wk 64 | N | 11 20 wks off epag | 7.0 | 6.9 | 50 0.62 mg/kg | 75 0.88 mg/kg | 3 Frequent episodes of epistaxis, at least once per month lasting ≥30 min. On epag, fewer episodes that rarely lasted 10 min | 0 | 0 |
| 7 | 16 | 154 wk 152 | Y | 154 wk 152 | 5.7 | 6.3 | 35 1.9 mg/kg | 75 2.8 mg/kg | 3 Frequent bruising in addition to ICH and very occasional epistaxis that lasted ≥15 min. On epag, no bleeding with increased dose to 75 mg at month 18 | No platelet transfusions required while on epag (had required them prior to therapy for ICH) | Discontinued daily amicar while on epag |
| 8* | 44 | 98 wks 82 and 111 | Y | 47 wk 209 | 5.4 | 6.2 | 9 0.7 mg/kg | 75 3.3 mg/kg | 3 Substantial bruising, oral bleeding, and epistaxis. On epag, fewer episodes of epistaxis and decreased bruising | Less frequent platelet transfusions required (only 1 not related to surgical procedures) | No change in WAS/XLT tx |
| 9 | 20 | 134 wk 126 | Y | 134 wk 126 | 9 | 7.1 | 20 1.6 mg/kg | 60 3.8 mg/kg | 3 Frequent bruising, constant petechiae, and one major bleed when hitting lip. On epag, no bruising or bleeding. Pt is more energetic while on epag | 0 | 0 IVIG ×1 while on epag when pt had a viral infection |
| 12† | 14 | 283 wk 22 | N | 128 24 wks off epag | 6.2 | 5.8 | 25 1.6 mg/kg | 75 5 mg/kg | 2 Before prednisone and epag, bruising and other bleeding. No bleeding on prednisone alone. With addition of epag, no bleeding, including when prednisone was subsequently discontinued | 0 | Discontinued daily prednisone (0.2 mg/kg) while on epag |
| Nonresponders | |||||||||||
| 10 | 9 | 166 wk 6 (after platelet trans-fusion) | N | Lost to follow-up | 7.2 | 8.2 | 50 1.3 mg/kg | 75 2.1 mg/kg | 1 | Frequent platelet transfusions required before and during epag therapy | 0 |
| 11 | 15 | 37 wk 187 | Y | 17 wk 198 | 7.7 | 8.3 | 25 1.1 mg/kg | 75 2.8 mg/kg | 3 Bruising and other minor bleeding. On epag, no bleeding. Pt is more energetic on epag. | Does not require platelet transfusions (had required weekly platelet transfusions prior to epag) | 0 |
| 13 | 13 | 55 wk 2 | N | 30 58 wks off epag, while on romiplostim | 9.5 | 8.2 | 50 weight n/a | 75 weight n/a | 3 On epag, slightly less skin and mucous membrane bleeding | 0 | 0 |
Epag, eltrombopag; ICH, intracranial hemorrhage; n/a, not available; Pt, patient; tx, treatment with eltrombopag.
Continuing IVIG every 3 to 4 weeks as prophylaxis for infection.
In view of the uncertainty regarding response to eltrombopag, it was decided to continue the prednisone and add eltrombopag with the plan of discontinuing the prednisone if the eltrombopag elevated the platelet count.