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Phase II study of efficiency of peripheral blood stem cell harvest using low-dose cyclophosphamide plus G-CSF for POEMS syndrome

LDCY-POEMS study

14

14 patients with POEMS syndrome were enrolled in this single-center, single-arm study. 7 males and 7 females. The median age was 53 years (47-59). PS was 1-2 in 8 patients (57.1%), 3-4 in 5 patients (42.9%), and the median serum VEGF was 4652 pg/mL (1990-6390).

This study was conducted under ethical guidelines based on the Declaration of Helsinki. Participants obtained informed consent at Chiba University Hospital. Written informed consent was obtained from all 14 patients. There were no dropouts or discontinuations. All participants received the planned dose of cyclophosphamide, and from the next day,10 ug/kg of G-CSF was subcutaneously administered daily. Stem cells were collected using Spectra Optia in all cases, and stem cells were measured using FACS. The harvested stem cells were cryopreserved in a freezer using 10% DEMSO. Pretreatment was performed using melphalan two days before transplantation, and stem cell transplantation was performed. As for the engraftment, the day when the neutrophil count of 500/microL or more and the platelet count of 20,000/microL or more was confirmed at 3 points, was determined as the day of engraftment.

No disease or adverse event during the study period was observed.

The target cell dose was 2*10e6/kg of CD34-positive cells. Twelve out of 14 patients (85.7%, 95%CI 57.2-98.2) reached the target cell number in a one-day procedure. All patients underwent autologous peripheral blood stem cell transplantation and survived. The median CD34+cells collected on day 1 was 6.31*10e6/kg (range, 0.69-14.13). Two cases in which a one-day procedure did not reach the target cell number were collected again the following day. In one case, the collection treatment could secure the target number of cells on the second day, but the target number could not be secured in one case. The results of the secondary endpoints, the days of engraftment, included median neutrophil engraftment days of 15 (14-19) days and platelet engraftment median days of 14.5 (14-18) days. There were no Grade 3 events, and our stem cell collection protocol was well tolerated.

The CY dose used for PBSC mobilization in patients with POEMS syndrome varied widely (range1-7g/mm). In this study, we analyzed the importance of cyclophosphamide dose intensity on PBSCH mobilization comparing with previous report. Of 85.7% were successfully mobilized stem cells. Low dose CY regimen decreased infectious complications and morbidity

Oct. 14, 2023

http://www.jshem.or.jp/85/

No

None

https://jrct.niph.go.jp/latest-detail/jRCTs031180421

Sakaida Emiko

Department of Hematology, Chiba University Hospital

1-8-1 Inohana, Chuo-ku, Chiba

esakaida@faculty.chiba-u.jp

Mimura Naoya

Department of Hematology, Chiba University Hospital

1-8-1 Inohana, Chuo-ku, Chiba

+81-43-222-7171

naoyamimura@chiba-u.jp

Complete

July. 22, 2015

Interventional

single arm study

open(masking not used)

no treatment control/standard of care control

single assignment

treatment purpose

1) Patients diagnosed with multiple myeloma based on IWMG criteria 2) Bone marrow plasma cells under 5% after chemotherapy 3) Lenalidomide treatment under 4 cycles 4) More than 4 weeks after the beginning day of the last chemotherapy 5) Age 20-70 6) Within 14 days prior to registration Neutrophil over 1,000/mm3 Hemoglobin over 8.0g/dlPlatelet over 100,000/mm3 GOT, GPT under 2.5 times the upper limit of normal level Total bilirubin under 2.0mg/dl Creatinine under 2.0mg/dl ECG WNL or trivial change without any symptoms SpO2 over 95% 7) More than 3 months survival expected 8) Informed consent obtained

1) Pregnant or lactating females. Patients who cannot do contraception.
2) Complications of active other cancers.
3) Severe psychological disorder
4) HBsAg positive or HBV-DNA positive.
5) HIV-Ab positive.
6) HCV-Ab positive.
7) Plasma cells in the peripheral blood.
8) Severe drug allergy.
9) Active infection.
10) Any other problems regarded as disqualification by doctors.

Both

POEMS syndrome

Treatment by cyclophosphamide, mesna, graniserton, and lenograstim. Autologous peripheral blood stem cell harvest (auto-PBSCH)

POEMS syndrome

PBSCH, cyclophosphamide, G-CSF

Success rate of PBSCH to obtain 2 times10^6/kg CD34 positive cells for a transplantation

effectiveness Day of engraftment safetyFrequency of adverse events (any grade/grade 3 and more), frequency of adverse response against drugs

+81-43-226-2616

Feb. 20, 2019

none