Contributions
Abstract: PB1488
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Thrombopoietin analogs (aTPO) are used due to effectiveness in megakaryocytes stimulus and bone marrow multipotent hematopoietic progenitor cells. Thus, are widely used as second row treatment in patients with refractory medullary aplasia. Currently, it could be used as first contact treatment with immunosuppressive therapy enhacing in order to increase the response to treatment.
Aims
Analyze of Romiplostim use after inmunosuppressive therapy in patients with severe medullary aplasia.
Methods
Data analysis of all the patients diagnosed with medullary aplasia between 2019 - 2021 and with Romiplostim treatment has been done taking into account the effiency of the treatment in two terms:
- Parcially response to treatment: neutrophils higher than 0.5x10e9/L, platelets higher than 20x10e9/L and haemoglobin higher than 8 g/dL.
- Complete response to treatment: neutrophils higher than 1.5x10e9/L, platelets higher than 150x10e9/L and haemoglobin higher than 12-13 g/dL.
Results
In table 1 below, characteristics of 4 patients treated with Romiplostim has been shown. Three of them had platelets lower than 20x10e9/L with transfusional dependency and neutrophils lower than 1x10e9/L. All of them had have immunosuppressive therapy + Eltrombopag without response. Two of them had refraction to platelets transfusion. One of them only had been received previous treatment with Eltrombopag. After 6,5 months of tracking, all the patients treated with Romiplostim achieve parcially response to treatment without serious complications.
Pacient | Age | Gender | Diagnosis | Immunosuppressive therapy | Refractariedad plaquetaria | Eltrombopag treatment |
1 | 73 | F | Severe medullary aplasia | YES | YES | YES (No response) |
2 | 52 | F | Severe medullary aplasia | YES | YES | YES (No response) |
3 | 68 | M | Severe medullary aplasia | YES | NO | YES (No response) |
4 | 33 | M | Severe medullary aplasia | YES | NO | NO |
Table 1. Characteristics of patients treated with Romiplostim.
Pacient | Start of Romiplostim | Response | Complications | At present |
1 | 22/05/2019
Hb: 9.5 g/dL N: 0.7x10e9/L Pla: 21x10e9/L (transfused)
| At 2 months at doses of 8 mcg/Kg
| NO | Sustained partial response at doses of 10 mcg/Kg
Hb: 12.9 g/dL N: 1.7x10e9/L Pla: 150x10e9/L
|
2 | 31/01/2020
Hb: 10.1 g/dL N: 0.98x10e9/L Pla: 41x10e9/L (transfundidas) | At 4 months at doses of 7 mcg/Kg
| NO | Sustained partial response at doses of 7 mcg/Kg
Hb: 9.4 g/dL N: 2.34x10e9/L Plaq: 152x10e9/L
|
3 | 23/10/2020
Hb: 11 g/dL N: 0.59x10e9/L Pla: 26x10e9/L (transfundidas)
| At 8 months at doses of 10 mcg/Kg | NO | Sustained partial response at doses of 10 mcg/Kg
Hb: 12.1 g/dL N: 1.72x10e9/L Pla: 50x10e9/L
|
4 | 23/09/20
Hb: 7.8 g/dL N: 0.79x10e9/L Pla: 18x10e9/L
| At 2 months at doses of 6 mcg/Kg | NO | Sustained partial response at doses of 8 mcg/Kg.
Hb: 13,4 g/dL N: 1,87x10e9/L Pla: 66x10e9/L
|
Table 2. Results of the use of Romiplostim in medullary aplasia in our hospital.
Conclusion
Adjuvant use of Romiplostim with patients without immunosuppressive therapy response must be taken into account. This option decrease transfunctional needs and improve all the blood series (hemoperipheral level) without serious complications.
Keyword(s): Bone marrow failure, Thrombopoietin (TPO)
Abstract: PB1488
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Thrombopoietin analogs (aTPO) are used due to effectiveness in megakaryocytes stimulus and bone marrow multipotent hematopoietic progenitor cells. Thus, are widely used as second row treatment in patients with refractory medullary aplasia. Currently, it could be used as first contact treatment with immunosuppressive therapy enhacing in order to increase the response to treatment.
Aims
Analyze of Romiplostim use after inmunosuppressive therapy in patients with severe medullary aplasia.
Methods
Data analysis of all the patients diagnosed with medullary aplasia between 2019 - 2021 and with Romiplostim treatment has been done taking into account the effiency of the treatment in two terms:
- Parcially response to treatment: neutrophils higher than 0.5x10e9/L, platelets higher than 20x10e9/L and haemoglobin higher than 8 g/dL.
- Complete response to treatment: neutrophils higher than 1.5x10e9/L, platelets higher than 150x10e9/L and haemoglobin higher than 12-13 g/dL.
Results
In table 1 below, characteristics of 4 patients treated with Romiplostim has been shown. Three of them had platelets lower than 20x10e9/L with transfusional dependency and neutrophils lower than 1x10e9/L. All of them had have immunosuppressive therapy + Eltrombopag without response. Two of them had refraction to platelets transfusion. One of them only had been received previous treatment with Eltrombopag. After 6,5 months of tracking, all the patients treated with Romiplostim achieve parcially response to treatment without serious complications.
Pacient | Age | Gender | Diagnosis | Immunosuppressive therapy | Refractariedad plaquetaria | Eltrombopag treatment |
1 | 73 | F | Severe medullary aplasia | YES | YES | YES (No response) |
2 | 52 | F | Severe medullary aplasia | YES | YES | YES (No response) |
3 | 68 | M | Severe medullary aplasia | YES | NO | YES (No response) |
4 | 33 | M | Severe medullary aplasia | YES | NO | NO |
Table 1. Characteristics of patients treated with Romiplostim.
Pacient | Start of Romiplostim | Response | Complications | At present |
1 | 22/05/2019
Hb: 9.5 g/dL N: 0.7x10e9/L Pla: 21x10e9/L (transfused)
| At 2 months at doses of 8 mcg/Kg
| NO | Sustained partial response at doses of 10 mcg/Kg
Hb: 12.9 g/dL N: 1.7x10e9/L Pla: 150x10e9/L
|
2 | 31/01/2020
Hb: 10.1 g/dL N: 0.98x10e9/L Pla: 41x10e9/L (transfundidas) | At 4 months at doses of 7 mcg/Kg
| NO | Sustained partial response at doses of 7 mcg/Kg
Hb: 9.4 g/dL N: 2.34x10e9/L Plaq: 152x10e9/L
|
3 | 23/10/2020
Hb: 11 g/dL N: 0.59x10e9/L Pla: 26x10e9/L (transfundidas)
| At 8 months at doses of 10 mcg/Kg | NO | Sustained partial response at doses of 10 mcg/Kg
Hb: 12.1 g/dL N: 1.72x10e9/L Pla: 50x10e9/L
|
4 | 23/09/20
Hb: 7.8 g/dL N: 0.79x10e9/L Pla: 18x10e9/L
| At 2 months at doses of 6 mcg/Kg | NO | Sustained partial response at doses of 8 mcg/Kg.
Hb: 13,4 g/dL N: 1,87x10e9/L Pla: 66x10e9/L
|
Table 2. Results of the use of Romiplostim in medullary aplasia in our hospital.
Conclusion
Adjuvant use of Romiplostim with patients without immunosuppressive therapy response must be taken into account. This option decrease transfunctional needs and improve all the blood series (hemoperipheral level) without serious complications.
Keyword(s): Bone marrow failure, Thrombopoietin (TPO)