Supplementary material Safety and Effectiveness of Plerixafor for Peripheral Blood Stem Cell Mobilization in Autologous Stem Cell Transplantation: Results of a Post-Marketing Surveillance Study Journal:

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Supplementary material

Safety and Effectiveness of Plerixafor for Peripheral Blood Stem Cell Mobilization in

Autologous Stem Cell Transplantation: Results of a Post-Marketing Surveillance Study Journal: Drugs Real World Outcomes

Authors: Nobuhiro Tsukada,¹ Momoko Nishikori,² Hiroaki Goto,³ Rie Kanamori,⁴ Satoshi

Nishina,⁵Takashi Seto,⁵ Shinsuke Iida⁶

Affiliations: ¹Department of Hematology, Japanese Red Cross Medical Center, Tokyo;

2Department of Hematology/Oncology, Graduate School of Medicine, Kyoto University, Kyoto;

3Division of Hematology/Oncology, Kanagawa Children's Medical Center, Kanagawa; ⁴Sanofi Genzyme Medical, Oncology Medical, Sanofi K.K., Tokyo; ⁵Medical Affairs, Post-Authorization Regulatory Studies, Sanofi K.K., Tokyo; ⁶Department of Hematology & Oncology, Nagoya City University Institute of Medical and Pharmaceutical Sciences, Nagoya

Corresponding Author: Shinsuke Iida, Department of Hematology & Oncology, Nagoya City University Institute of Medical and Pharmaceutical Sciences, Nagoya, Japan

Email: iida@med.nagoya-cu.ac.jp

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Supplementary Tables

Table S1. Proportion of Patients Achieving a CD34+ Cell Yield ≥2 x 10⁶cells/kg by Disease Subtype (Effectiveness Analysis Set)

Disease Subtype

MM ML Other^*

Total, n 310 327 72

Proportion of patients reaching a CD34+ cell yield of

≥2 x 10⁶cells/kg ≤2 days, n (%)

229 (73.9) 204 (62.4) 50 (69.4)

Proportion of patients reaching a CD34+ cell yield of

≥2 x 10⁶cells/kg ≤4 days, n (%)

235 (75.8) 215 (65.7) 54 (75.0)

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Median (95 % CI) number of days to reach a CD34+

cell yield of ≥2 x 10⁶cells/kg

1.0 (−) 2.0 (1.0, 2.0) 1.0 (−)

Days reaching a CD34+ cell yield of

≥2 x 10⁶cells/kg, n (%)

1 188 (60.6) 153 (46.8) 46 (63.9)

2 229 (73.9) 204 (62.4) 50 (69.4)

3 235 (75.8) 214 (65.4) 54 (75.0)

4 235 (75.8) 215 (65.7) 54 (75.0)

OR (95% CI) − 0.81 (0.68−0.98) 0.99 (0.74−1.33)

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protein, skin changes. *Other includes AL amyloidosis, neuroblastoma, POEMS syndrome, plasma cell leukemia, medulloblastoma, and Ewing’s sarcoma.

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Table S2. Proportion of Patients Achieving a CD34+ Cell Yield of ≥2 x 10⁶cells/kg by CD34+ Cell Count Prior to Study Drug Administration

Days to Reach a CD34+ Cell Yield of

≥2 x 10⁶cells/kg, n (%)

Days to Reach a CD34+ Cell Yield of ≥2 x 10⁶cells/kg,

Proportion of Patients Reaching a

CD34+ cell Yield of

≥2 x 10⁶cells/kg ≤4 days, n (%)

Proportion of Patients Reaching a CD34+ Cell Yield of

≥2 x 10⁶cells/kg ≤2 days, n (%)

n 1 2 3 4 Median

(95% CI)

HR (95% CI)

Baseline CD34+

Cell Count, cells/μL

0─<1

13 1 (7.7) 3 (23.1) 5 (38.5) 5 (38.5) 3.0 (2.0─9.0) ─ 5 (38.5) 3 (23.1)

1─<2

13 2 (15.4) 6 (46.2) 8 (61.5) 8 (61.5) 3.0 (2.0─3.0) 1.69 (0.58─4.90)

8 (61.5) 6 (46.2)

2─<5

56 32 (57.1) 44 (78.6) 45 (80.4) 45 (80.4) 1.0 (1.0─2.0) 3.00 45 (80.4) 44 (78.6)

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5─<10

53 36 (67.9) 43 (81.1) 44 (83.0) 44 (83.0) 1.0 (─) 3.25 (1.37─7.69)

44 (83.0) 43 (81.1)

10─<20

42 33 (78.6) 36 (85.7) 36 (85.7) 36 (85.7) 1.0 (─) 3.76 (1.57─9.03)

36 (85.7) 36 (85.7)

≥20

50 40 (80.0) 42 (84.0) 42 (84.0) 42 (84.0) 1.0 (─) 3.80 (1.60─9.04)

42 (84.0) 42 (84.0)

Unknown

12 8 (66.7) 9 (75.0) 9 (75.0) 9 (75.0) 1.0 (1.0─2.0) 3.18 (1.12─9.01)

9 (75.0) 9 (75.0)

Not

implemented 470 235 (50.0)

300 (63.8)

314 (66.8)

315 (67.0)

1.0 (1.0─2.0) 2.35 (1.04─5.31)

315 (67.0) 300 (63.8)

Data is presented as n (%) unless otherwise specified. OR, odds ratio.

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Table S3. Proportion of Patients Achieving a CD34+ Cell Yield of ≥2 x 10⁶cells/kg in Patients With Prior HSC Collection Versus No Prior HSC Collection by CD34+ Cell Count Prior to Study Drug Administration

n CD34+ Cell Count Prior to Study Drug Administration

Days to Reach a CD34+ Cell Yield of ≥2 x 10⁶cells/kg, n (%)

Proportion of Patients Reaching a CD34+ Cell Yield of ≥2 x 10⁶cells/kg

≤4 days, n (%)

Proportion of Patients Reaching a CD34+ Cell Yield of ≥2 x 10⁶cells/kg

≤2 days, n (%)

n Mean

(SD)

Median (range)

1 2 3 4

Total 709 227 25.3

(69.6)

7.0 (0.0─668.0) 387 (54.5) 483 (68.1)

503 (70.9)

504 (71.1)

504 (71.1) 483 (68.1)

No Prior HSC Collection

556 191 25.0

(69.8)

8.0 (0.0─668.0) 347 (62.4) 423 (76.1)

436 (78.4)

437 (78.6)

437 (78.6) 423 (76.1)

Baseline CD34+

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0─<1

7 7 0.4 (0.5) 0.1 (0.0─1.0) 0 2 (28.6) 3 (42.9) 3 (42.9) 3 (42.9) 2 (28.6)

1─<2

10 10 1.1 (0.3) 1.0 (1.0─1.9) 2 (20.0) 6 (60.0) 7 (70.0) 7 (70.0) 7 (70.0) 6 (60.0) 2─<5

42 42 3.1 (0.9) 3.0 (2.0─4.9) 27 (64.3) 37 (88.1) 38 (90.5)

38 (90.5) 38 (90.5) 37 (88.1)

5─<10

48 48 6.8 (1.3) 6.7 (5.0─9.2) 34 (70.8) 41 (85.4) 41 (85.4)

41 (85.4) 41 (85.4) 41 (85.4)

10─<20

41 41 13.8 (3.3) 12.7

(10.0─19.0)

33 (80.5) 36 (87.8) 36 (87.8)

36 (87.8) 36 (87.8) 36 (87.8)

≥20

43 43 87.1

(129.9)

34.0 (20.0─668.0)

37 (86.0) 38 (88.4) 38 (88.4)

38 (88.4) 38 (88.4) 38 (88.4)

Unknown

9 0 ─ ─ 7 (77.8) 8 (88.9) 8 (88.9) 8 (88.9) 8 (88.9) 8 (88.9)

Not evaluated

356 0 ─ ─ 207 (58.1) 255 265 266 266 (74.7) 255 (71.6)

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(71.6) (74.4) (74.7)

Prior HSC Collection

153 36 26.8

(69.3)

3.5 (0.0─304.0) 40 (26.1) 60 (39.2) 67 (43.8)

67 (43.8) 67 (43.8) 60 (39.2)

Baseline CD34+

0─<1

6 6 0.2 (0.3) 0.2 (0.0─0.6) 1 (16.7) 1 (16.7) 2 (33.3) 2 (33.3) 2 (33.3) 1 (16.7)

1─<2

3 3 1.2 (0.2) 1.2 (1.0─1.3) 0 0 1 (33.3) 1 (33.3) 1 (33.3) 0

2─<5

14 14 3.2 (0.9) 3.0 (2.0─4.8) 5 (35.7) 7 (50.0) 7 (50.0) 7 (50.0) 7 (50.0) 7 (50.0) 5─<10

5 5 5.5 (0.5) 5.5 (5.0─6.0) 2 (40.0) 2 (40.0) 3 (60.0) 3 (60.0) 3 (60.0) 2 (40.0)

10─<20

1 1 10.1 (─) 10.1

(10.1─10.1)

0 0 0 0 0 0

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(118.2) (20.0─304.0) Unknown

3 0 ─ ─ 1 (33.3) 1 (33.3) 1 (33.3) 1 (33.3) 1 (33.3) 1 (33.3)

Not evaluated

114 0 ─ ─ 28 (24.6) 45 (39.5) 49

(43.0)

49 (43.0) 49 (43.0) 45 (39.5)

Data is presented as n (%) unless otherwise specified. HSC, hematopoietic stem cell.