Systematic literature review of treatments used for adult immune thrombocytopenia in the second‐line setting

Bylsma, Lauren C.; Fryzek, Jon P.; Cetin, Karynsa; Callaghan, Fiona; Bezold, Carla; Mehta, Bhakti; Wasser, Jeffrey S.

doi:10.1002/ajh.25301

Cited by 31 publications

(52 citation statements)

References 61 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

mentioning

confidence: 99%

“…Eight meta‐analyses assessed second‐line medical therapy in paediatric and adult patients with newly diagnosed, relapsed and persistent ITP (Cooper et al , ; Chugh et al , ; Feng et al , ; Wang et al , ; Elgebaly et al , ; Arai et al , ; Zhang et al , ; Bylsma et al , ). Among them, 4 (Cooper et al , ; Wang et al , ; Elgebaly et al , ; Zhang et al , ), 2 (Chugh et al , ; Feng et al , ) and 2 (Arai et al , ; Bylsma et al , ) meta‐analyses assessed efficacy of TPO‐RAs, monoclonal antibody and both, respectively. For the TPO‐RAs, 2 meta‐analyses (Wang et al , ; Elgebaly et al , ) combined paediatric and adult ITP patients, 1 (Wang et al , ) combined randomised controlled trials (RCTs) with observational studies, and the rest (Cooper et al , ; Elgebaly et al , ; Arai et al , ; Zhang et al , ; Bylsma et al , ) considered only RCTs.…”

mentioning

confidence: 99%

“…Among them, 4 (Cooper et al , ; Wang et al , ; Elgebaly et al , ; Zhang et al , ), 2 (Chugh et al , ; Feng et al , ) and 2 (Arai et al , ; Bylsma et al , ) meta‐analyses assessed efficacy of TPO‐RAs, monoclonal antibody and both, respectively. For the TPO‐RAs, 2 meta‐analyses (Wang et al , ; Elgebaly et al , ) combined paediatric and adult ITP patients, 1 (Wang et al , ) combined randomised controlled trials (RCTs) with observational studies, and the rest (Cooper et al , ; Elgebaly et al , ; Arai et al , ; Zhang et al , ; Bylsma et al , ) considered only RCTs. Three of them directly pooled the effects of eltrombopag and romiplostim individually or combined them as TPO‐RAs (Wang et al , ; Elgebaly et al , ; Bylsma et al , ), while 3 (Cooper et al , ; Arai et al , ; Zhang et al , ) indirectly pooled the effects of romiplostim relative to eltrombopag.…”

mentioning

confidence: 99%

“…For the TPO‐RAs, 2 meta‐analyses (Wang et al , ; Elgebaly et al , ) combined paediatric and adult ITP patients, 1 (Wang et al , ) combined randomised controlled trials (RCTs) with observational studies, and the rest (Cooper et al , ; Elgebaly et al , ; Arai et al , ; Zhang et al , ; Bylsma et al , ) considered only RCTs. Three of them directly pooled the effects of eltrombopag and romiplostim individually or combined them as TPO‐RAs (Wang et al , ; Elgebaly et al , ; Bylsma et al , ), while 3 (Cooper et al , ; Arai et al , ; Zhang et al , ) indirectly pooled the effects of romiplostim relative to eltrombopag. For the 2 direct meta‐analyses on monoclonal antibody, 5 (Chugh et al , ) and 7 (Feng et al , ) RCTs comparing rituximab with placebo or standard treatments were pooled.…”

mentioning

confidence: 99%

See 3 more Smart Citations

Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

et al. 2019

View full text Add to dashboard Cite

Persistent immune thrombocytopenia (ITP) patients require second-line treatments, for which information on clinical outcomes are lacking. A systematic review and network meta-analysis (NMA) were conducted. Only randomised controlled trials (RCT) of second-line drugs in adult persistent ITP patients with platelet response, platelet count, any bleeding or serious adverse events (SAE) outcome were eligible. Twelve RCTs (n = 1313) were included in NMA. For platelet response outcome, eltrombopag and romiplostin were the best relative to placebo; the former had a non-significant advantage [risk ratio (RR) = 1Á10 (95% confidence interval: 0Á46, 2Á67)]. Both treatments were superior to rituximab and recombinant human thrombopoietin (rhTPO)+rituximab, with corresponding RRs of 4Á56 (1Á89, 10Á96) and 4Á18 (1Á21, 14Á49) for eltrombopag; 4Á13 (1Á56, 10Á94) and 3Á79 (1Á02, 14Á09) for romiplostim. For platelet count, romiplostim ranked highest, followed by eltrombopag, rhTPO+rituximab, and rituximab. For bleeding, rituximab had lowest risk, followed by eltrombopag and romiplostim. For SAEs, rhTPO+rituximab had highest risk, followed by rituximab, eltrombopag and romiplostim. From clustered ranking, romiplostim had the best balance between short-term efficacy and SAEs, followed by eltrombopag. In conclusion, romiplostim and eltrombopag may yield high efficacy and safety. Rituximab may not be beneficial due to lower efficacy and higher complications compared with the thrombopoietin receptor agonists. RCTs with long-term clinical outcomes are required.

show abstract

mentioning

confidence: 99%

mentioning

confidence: 99%

mentioning

confidence: 99%

mentioning

confidence: 99%

See 2 more Smart Citations

Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

et al. 2019

View full text Add to dashboard Cite

show abstract

“…Second, splenectomy is now frequently delayed with the increased preference on rituximab and thrombopoietin-receptor agonists. 9,10 If ITP patients do not respond to other therapies, splenectomy would be less likely to be effective in this setting. The IWG consensus report does not stipulate patient failure to multiple ITP-specific therapies or relapse.…”

Section: Introductionmentioning

confidence: 99%

A prospective, multicenter study of low dose decitabine in adult patients with refractory immune thrombocytopenia

et al. 2019

View full text Add to dashboard Cite

We conducted a prospective, multicenter study to evaluate the efficacy and safety of low-dose decitabine in adult patients with refractory immune thrombocytopenia.Adult patients who did not respond to, did not tolerate, or were unwilling to undergo splenectomy, with either a baseline platelet count less than 30 × 10 9 /L or the presence of bleeding symptoms and further need of ITP-specific treatments, were enrolled. Patients received decitabine at 3.5 mg/m 2 intravenously for three consecutive days per cycle, for three cycles with a four-week interval between cycles. All patients were assessed every week during the first 12 weeks and at four-week intervals thereafter. We screened 49 patients for eligibility. Four patients were excluded and 45 received decitabine. At the end of decitabine treatment, complete response was achieved in eight patients (17.78%), and partial response was achieved in 15 patients (33.33%). The median time to initial response was 28 days (range, 14-70 days). Furthermore, seven relapsed patients received decitabine retreatment and all showed platelet response, including one complete response and six partial responses. Sustained response rates at 6, 12 and 18 months were 44.44% (20/45), 31.11% (14/45) and 20.0% (9/45), respectively. For responders, immune thrombocytopenia-related symptoms, fatigue, psychological health, fear, and overall quality of life were significantly improved. Adverse events were observed in 13 (28.89%) patients. No serious adverse events were recorded. In conclusion, low dose decitabine is potentially effective and safe in the management of adults with refractory immune thrombocytopenia. This trial is registered with clinicaltrials.gov identifier: NCT01568333.

show abstract

Quality of life is an important indication for second‐line treatment in children with immune thrombocytopenia

Shimano

Neunert

Bussel

et al. 2021

Pediatric Blood & Cancer

View full text Add to dashboard Cite

Background The decision to initiate second‐line treatment in children with immune thrombocytopenia (ITP) is complex and involves many different factors. Methods In this prospective, observational, longitudinal cohort study of 120 children from 21 centers, the factors contributing to the decision to start second‐line treatments for ITP were captured. At study entry, clinicians were given a curated list of 12 potential reasons the patient required a second‐line treatment. Clinicians selected all that applied and ranked the top three reasons. Results Quality of life (QOL) was the most frequently cited reason for starting a second‐line therapy. Clinicians chose it as a reason to treat in 88/120 (73%) patients, as among the top three reasons in 68/120 (57%), and as the top reason in 32/120 (27%). Additional factors ranked as the top reason to start second‐line treatment included severity of bleeding (22/120, 18%), frequency of bleeding (19/120, 16%), and severity of thrombocytopenia (18/120, 15%). Patients for whom QOL (p = .006) or sports participation (p = .02) were ranked reasons were more likely to have chronic ITP, whereas those for whom severity (p = .003) or frequency (p = .005) of bleeding were ranked reasons were more likely to have newly diagnosed or persistent ITP. Parental anxiety, though rarely the primary impetus for treatment, was frequently cited (70/120, 58%) as a contributing factor. Conclusion Perceived QOL is the most frequently selected reason pediatric patients start second‐line therapies for ITP. It is critical that studies of treatments for childhood ITP include assessments of their effects on QOL.

show abstract

Systematic literature review of treatments used for adult immune thrombocytopenia in the second‐line setting

Cited by 31 publications

References 61 publications

Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

Treatment efficacy for adult persistent immune thrombocytopenia: a systematic review and network meta‐analysis

A prospective, multicenter study of low dose decitabine in adult patients with refractory immune thrombocytopenia

Quality of life is an important indication for second‐line treatment in children with immune thrombocytopenia

Contact Info

Product

Resources

About