internal medicine
Contributions
Type: Publication Only
Background
The classical therapeutic approach for Immune Thrombocytopenic Purpura (ITP), provides the use of corticosteroids and intravenous immunoglobulins (IVIg) as first line treatment and, in case of refractoriness, the splenectomy as second line therapy. Since more than ten years other two drug regimens with Rituximab and TPO-ra, are available for the ITP treatment but their precise location in the stages of the disease is still debated.
Aims
Goal of this study was to evaluate, retrospectively, the use and efficacy of Rituximab and TPO-ra in the real-life practice of ITP to define the best time for their use.
Methods
The response to Rituximab and TPO-ra was investigated in patients treated (21 and 15 ITP patients respevtively) between May 2004 and February 2015 at our ward. The response was evaluated in terms of efficacy (CR*= Complete Response > 100 plt x 109L, PR= Partial Response > 50 plt x 109L, MR= Minimal Response > 30 plt x 109L, NR= None Response < 30 plt x 109L or no change from the baseline platelet count) and duration (SR**= Sustained Response > 6 months, PeR= Persistent Response > 3 months, TR= Transitory Response < 3 months).
Results
The results are summarized in Table 1 and Table 2. Overall response to Rituximab was 62% and to TPO-ra was 67%. The response rate in Rituximab group decreases significantly if it was used as second line of treatment (69%) rather than as third (23%) or fourth (8%) line. Our results don’t show the same trend for the TPO-ra group where the response seems to be independent from the time of use and from the previous treatments (40% of rat response in second line; 40% in the third line; 20% in the fourth line).
Table 1. Efficacy and duration of the response
| Rituximab | TPO-ra |
Total treated patients | 21 | 15 |
Complete Response* (CR) n (%) | 11 (52%) | 10 (67%) |
1 (5%) | 0 | |
1 (5%) | 0 | |
Sustained Response (SR)** n (%) | 6 (47% of responders) | 6 (60% of responders) |
5 (38% of responders) | 3 (30% of responders) | |
2 (15% of responders) | 1 (10 % of responders) | |
None Response (NR) n (%) | 8 (38%) | 5 (33%) |
Response Onset-time (weeks) | 2 (1; 8) | 1 (1; 2) |
Table 2. Relationship between response and previous treatments
| Rituximab | TPO-ra | ||
Responders | Non-responders | Responders | Non-responders | |
Total patients n (%) | 13 (100%) | 8 (100%) | 10 (100%) | 50 (100%) |
Second line n (%) | 9 (69%) | 5 (63%) | 4 (40%) | 1 (20%) |
Third line n (%) | 3 (23%) | 3 (37%) | 4 (40%) | 1 (20%) |
Fourth line n (%) | 1 (8%) | 0 | 2 (20%) | 3 (60%) |
Splenectomy n (%) | 3 (23%) | 4 (50%) | 3 (30%) | 2 (40%) |
Plts (n x 10.9L) before treatment | 5 (1; 74) | 12 (3; 50) | 12 (1; 30) | 4 (2; 5) |
Summary
The overall response is than clearly higher when Rituximab was used in an early stage of ITP showing a marked trend to inefficacy in the chronic and multi-refractory phases. Actually we can’t say the same thing for TPO-ra. Prospective and multicenter studies about the use of these drugs in acute ITP should be performed to validate these results.
Keyword(s): ITP, Rituximab
Type: Publication Only
Background
The classical therapeutic approach for Immune Thrombocytopenic Purpura (ITP), provides the use of corticosteroids and intravenous immunoglobulins (IVIg) as first line treatment and, in case of refractoriness, the splenectomy as second line therapy. Since more than ten years other two drug regimens with Rituximab and TPO-ra, are available for the ITP treatment but their precise location in the stages of the disease is still debated.
Aims
Goal of this study was to evaluate, retrospectively, the use and efficacy of Rituximab and TPO-ra in the real-life practice of ITP to define the best time for their use.
Methods
The response to Rituximab and TPO-ra was investigated in patients treated (21 and 15 ITP patients respevtively) between May 2004 and February 2015 at our ward. The response was evaluated in terms of efficacy (CR*= Complete Response > 100 plt x 109L, PR= Partial Response > 50 plt x 109L, MR= Minimal Response > 30 plt x 109L, NR= None Response < 30 plt x 109L or no change from the baseline platelet count) and duration (SR**= Sustained Response > 6 months, PeR= Persistent Response > 3 months, TR= Transitory Response < 3 months).
Results
The results are summarized in Table 1 and Table 2. Overall response to Rituximab was 62% and to TPO-ra was 67%. The response rate in Rituximab group decreases significantly if it was used as second line of treatment (69%) rather than as third (23%) or fourth (8%) line. Our results don’t show the same trend for the TPO-ra group where the response seems to be independent from the time of use and from the previous treatments (40% of rat response in second line; 40% in the third line; 20% in the fourth line).
Table 1. Efficacy and duration of the response
| Rituximab | TPO-ra |
Total treated patients | 21 | 15 |
Complete Response* (CR) n (%) | 11 (52%) | 10 (67%) |
1 (5%) | 0 | |
1 (5%) | 0 | |
Sustained Response (SR)** n (%) | 6 (47% of responders) | 6 (60% of responders) |
5 (38% of responders) | 3 (30% of responders) | |
2 (15% of responders) | 1 (10 % of responders) | |
None Response (NR) n (%) | 8 (38%) | 5 (33%) |
Response Onset-time (weeks) | 2 (1; 8) | 1 (1; 2) |
Table 2. Relationship between response and previous treatments
| Rituximab | TPO-ra | ||
Responders | Non-responders | Responders | Non-responders | |
Total patients n (%) | 13 (100%) | 8 (100%) | 10 (100%) | 50 (100%) |
Second line n (%) | 9 (69%) | 5 (63%) | 4 (40%) | 1 (20%) |
Third line n (%) | 3 (23%) | 3 (37%) | 4 (40%) | 1 (20%) |
Fourth line n (%) | 1 (8%) | 0 | 2 (20%) | 3 (60%) |
Splenectomy n (%) | 3 (23%) | 4 (50%) | 3 (30%) | 2 (40%) |
Plts (n x 10.9L) before treatment | 5 (1; 74) | 12 (3; 50) | 12 (1; 30) | 4 (2; 5) |
Summary
The overall response is than clearly higher when Rituximab was used in an early stage of ITP showing a marked trend to inefficacy in the chronic and multi-refractory phases. Actually we can’t say the same thing for TPO-ra. Prospective and multicenter studies about the use of these drugs in acute ITP should be performed to validate these results.
Keyword(s): ITP, Rituximab