BACKGROUND

New drugs for pediatric immune thrombocytopenia have recently been developed; however, the practice patterns of the different treatments remain unclear in Japan.

METHODS

Using a national inpatient database, we analyzed recent trends in the treatment practice patterns of pediatric patients with immune thrombocytopenia.

RESULTS

From 2010 to 2021, the proportion of corticosteroid use did not change from 35.2 to 33.4%, whereas the proportion of intravenous immunoglobulin increased from 66.0 to 76.2% (P trend 0.003), and rituximab, romiplostim, and eltrombopag increased from 0.2 to 0.7%, 0.0 to 1.4%, and 0.3 to 7.5%, respectively, (P trend <0.001, 0.003, and <0.001, respectively). The proportion of no-treatment decreased from 20.2 to 6.5% (P trend <0.001). None of the patients had undergone splenectomy since 2016. Intravenous immunoglobulin and corticosteroids were mostly used at 1.0 g/kg and 2.0 mg/kg per day, respectively. We compared clinical practice patterns and outcomes between the only one-treatment and no-treatment groups. Intravenous immunoglobulin tended to be used in non-academic hospitals and younger patients; however, more risk of readmissions within 6 months occurred. Second-line treatment tended to be used in academic hospitals and older patients.

CONCLUSIONS

We observed an increasing trend in intravenous immunoglobulin use and second-line treatment, no change in corticosteroid use, and a decreasing trend in no-treatment approaches, using a national inpatient database. The hospital type might have affected the practice patterns. Clinicians might have chosen the optimal treatment for pediatric patients with immune thrombocytopenia considering several factors and patient backgrounds.

INTRODUCTION

Miller Fisher syndrome (MFS) is typically associated with a benign course and spontaneous recovery. However, immunotherapies such as intravenous immunoglobulin (IVIG) and plasma exchange (PE) are often administered for this condition because of the potential for clinical deterioration. We aimed to evaluate the real-world use of immunotherapies in MFS management using a nationwide Japanese database.

METHODS

We conducted a retrospective cohort study between April 2014 and March 2020 using the Japanese Diagnosis Procedure Combination database. Patient demographics and treatment modalities were described. Outcomes included length of hospital stay, in-hospital mortality, hospitalization cost, and activities of daily living defined by the Barthel index score.

RESULTS

We identified 1,595 patients with MFS. Of these, 999 (62.6%) received immunotherapy (including IVIG for 908 [56.9%] patients, intravenous methylprednisolone for 219 [13.7%] patients, and PE for 18 [1.8%] patients). In patients with and without immunotherapy, the median length of hospital stay was 18 and 12 days, in-hospital mortality was 0.7% and 0.2%, median total hospitalization costs were ¥1,660,200 and ¥549,375, and the proportions of Barthel index score of 95–100 at discharge were 75.3% and 75.2%, respectively.

CONCLUSION

Our findings highlight the widespread use of immunotherapies for MFS in Japan despite its generally favorable natural course.

BACKGROUND

Polypharmacy management represents a public health concern; however, intervention by community pharmacists remains limited. Therefore, we evaluated the effectiveness of community pharmacy-based educational intervention for improving polypharmacy management using interrupted time series analysis (ITSA).

METHODS

We conducted the ITSA of an educational intervention implemented from June to September 2021 across pharmacies under Medical System Network Co., Ltd., Japan. We analyzed pharmacy claims data from April 2020 to May 2024, with no missing values. The intervention included lectures and workshops delivered via cascade training. The primary and secondary outcomes were the number of Fee for Medication Adjustment Support 2 (FMAS2) and Fee for Medication Adjustment Support 1 (FMAS1) per 100,000 patients, respectively. FMAS2 is claimed when pharmacists propose reducing medications for patients taking six or more medications from multiple institutions, and FMAS1 is claimed when the proposal leads to actual reduction.

RESULTS

The study included 404 pharmacies with an average of 569,909 patients per month. After intervention, FMAS2 claims immediately increased by 3.82 per 100,000 people (95% confidence interval (CI): 1.23 to 6.41, p = 0.005), with no significant trend change. FMAS1 claims first increased by 1.29 per 100,000 people (95% CI: 0.49 to 2.09, p = 0.002) but decreased thereafter (95% CI: −0.298 to −0.130, p < 0.001).

CONCLUSIONS

Educational intervention increased FMAS2 and FMAS1 claims in the short term. However, the clinical impact of this intervention was small, and its long-term effectiveness is limited. Future efforts should focus on developing sustainable programs and follow-up systems.

The Fight Retinal Blindness! Registry is a prospectively-designed registry developed in 2009 that collects international data on treatment outcomes for eye diseases including neovascular age-related macular degeneration, diabetic macular edema and retinal vein occlusion. The validated and high-quality data have generated significant real-world evidence regarding clinically relevant issues, such as long-term visual outcomes, dosing regimens and practitioner variation. This paper outlines the Fight Retinal Blindness! Registry, highlighting its pros and cons, and introduces some findings from the registry.