Quantitative Research Report MPS1 Diagnoser Screening ... · 2 Contents 03 Research Design 07...

1

1

MPS1 Diagnoser Screening Market Research Report

IPSOS HEALTHCARE TURKEYOctober 2019

Quantitative Research Report

© 2019 Ipsos. All rights reserved. Contains Ipsos' Confidential and Proprietary information and may not be disclosed or reproduced without the prior written consent of Ipsos.

2

Contents

03 Research Design

07 Executive Summary

11 Patient Load

13 Cases & Experience

46 Information Level

55 Information Resources

3 © 2018 Ipsos.

RESEARCH DESIGN

4 © 2015 Ipsos.

Quantitative F2F Interviews

• LOI: 20 mins

• Specialty: Pediatric Endocrinologists

• Among physicians targeted by Sanofi

• On Jul – Sep 2019

• This report includes the findings by total, in breakdown of institutions due to their knowledge of MPS1 disease.

Sample Size :

• Total: 60 interviews with the Pediatric Endocrinologists in the list provided by Sanofi.

Market Research covers: • Awareness level of the physicians

regarding MPS1 disease and its’ diagnosis

• MPS1 patient load of the physicians

• Referral of patients• Diagnosis and treatment process of

the disease• Information resources

Objectives Approach Sample

Study Overview

5

23

16

15

3

1

1

1

ISTANBUL

ANKARA

IZMIR

ANTALYA

ADANA

ESKISEHIR

KAYSERI

Quick facts

City

Participants consisted of 60 Pediatric

Endocrinologists, who have been

working for 11 years on average

2529

6

UniversityR&TPrivate

Institution

type

G1 / G2 / G3 / G4 / G5

Title

8

30

10 12

Professor Specialist Associate SubspecialtyAssistant

Sample Distribution

6

Demography

Ankaran:16

Antalyan:3

Kayserin:1

İstanbuln:23

İzmirn:15

Adanan:1

TOTALn:60

Eskişehirn:1

7 © 2018 Ipsos.

EXECUTIVE SUMMARY

8 © 2015 Ipsos.

EXECUTIVE SUMMARY

• The physicians who were interviewed have seen that;

• 22% of the patients have growth failure

• 9% of the patients have Type 1 diabetes

• 21% of the patients have hypertiroidism

Patient Load

Case 1;

• 42% of the physicians claimed that they have seen patient(s) who fit the profile in the last year.

• 43% of the physicians suspected of Growth Hormone Deficiency which is followed by Thyroid / Hypothyroid Diseases with 25% at first. With the help of the additional information; physicians suspected of MPS1 (22%), Metabolic Diseases (18%), Skeleton / Bone Dysplasia (18%), and Turner Syndrome (17%)

• 48% of the physicians claimed that they would have treated the patient themselves. The rest would refer the patient mostly to Pediatric Metabolism or Pediatric Rheumatology. With the additional information treatment ratio decreased to 33%

• Physicians would have run Thyroid (42%) and Biochemical Tests (40%) w/o the additional information but 40% of them chose to refer the patient after they heard new info about the case

• Physicians assumed the disease could be related to Myopathy with a ratio of 62%

• Among the physicians who suspected of MPS1 Disease 45% of them explained it with Short Stature and 41% of them with Joint Contractures.

Cases & Experience

9 © 2015 Ipsos.

EXECUTIVE SUMMARY

Case 2;

• 10% of the physicians claimed that they have seen patient(s) who fit the profile in the last year.

• 42% of the physicians suspected of Rheumatismal Diseases which is followed by Metabolic Diseases (18%) at first. With the help of the additional information; physicians suspected of Metabolic Diseases (28%), MPS1 (25%), Lysosomal Storage Disease (22%)

• 13% of the physicians claimed that they would have treated the patient themselves. The rest would refer the patient mostly to Pediatric Rheumatology or Pediatric Metabolism. With the additional information treatment ratio is also 13%.

• Physicians would have refer the patient (55%) or run Blood Tests (18%) w/o the additional information but 40% of them chose to refer the patient after they heard new info about the case

• Physicians assumed the disease could be related to Arthrogryposis, Polymyositis, and Pompe Disease with 55%

• Among the physicians who suspected of MPS1 Disease 35% of them explained it with Short Stature and Corneal Opacity

Cases & Experience

10

EXECUTIVE SUMMARY

• Among total of physicians, 3% evaluate themselves as they know very well about MPS1 Disease and its treatment.

• University Hospital physicians are significantly less informed about the disease & treatment.

Information Level & Experience

• 43% of the total physicians would like to be informed about MPS1 Disease.

• The physicians would like to be informed about;

• Treatment (96%)

• Diagnostic tests (81%)

• Sub-types (81%)

• The physicians are mostly willing to be informed by Scientific Meetings, Congresses and Medical publications/reference books..

Information Resources

11 © 2018 Ipsos.

PATIENT LOAD

12

Total R&T Hospital University Hospital Private Hospital

Base 60 29* 25* 6**

Number of patients in 1 month 646 pts 802 pts 566 pts 227 pts

Patients with growth failure 22% (145 pts) 23% (186 pts) 21% (121 pts) 23% (52 pts)

Patients with type 1 diabetes 9% (57 pts) 8% (64 pts) 10% (59 pts) 9% (20 pts)

Patients with hypertiroidism 21% (135 pts) 22% (172 pts) 20% (116 pts) 15% (35 pts)

Base: All Physicians. G6. Son bir ayda toplam kaç hasta gördüğünüzü söyler misiniz?G7. Son bir ayda kaç ................. hastası gördüğünüzü söyler misiniz?

Patient Load

*Low base**Very low base

Institution Breakdown

13 © 2018 Ipsos.

CASES & EXPERIENCE

14

Case 1 - Information

Clinical Picture Medical Record

• 6 years old girl• Has a growth failure from age 1 for her age and gender• Her height is less then the 3rd percentile and it is below

the potential genetic target• Her nutritional status is good• Normal neurological evaluation• Hair on the face and arms (hirsutism)

• Was born on time and with normal birth scales• Underwent bilateral inguinal hernia surgery at 2 months of age• Recurrent upper respiratory infections and snoring history• Recurrent abdominal pain reported from the age of entering

the social environment (3 years old)• Slight joint stiffness in both hands and fingers

Base: All Physicians. A0. Son 12 ay içerisinde bu profile uygun bir hasta gördünüz mü? A0x. Son 12 ay içerisinde bu profile uygun kaç hasta gördüğünüzü söyler misiniz?


Base 60 29* 25* 6**

Yes / No

Base of physicians who say “Yes” 25* 14* 9** 2**

Patients who fit the profile in Case 1

17 pts 25 pts 5 pts 12 pts

42% 58% 48% 52% 36% 64% 33% 67%



Yes: I’ve seen patient(s) who fit the profile / No: I haven’t seen any patient who fit the profile

15


Base 60 29* 25* 6**

Growth Hormone Deficiency

Thyroid / Hypothyroid Diseases

Mucopolysaccharidosis I (MPS I)

Hypochondroplasia / Idiopathic Short Stature

Metabolic Diseases

Rheumatismal Diseases

Celiac Disease

Turner Syndrome

DSD (Disorder Of Sex Development)

Constitutional Short Stature Due To Familial Hypertrophyse

Case 1 - Suspected Diseases at First


43%

25%

17%

15%

12%

10%

10%

8%

7%

7%

27%

8%

8%

7%

5%

8%

2%

7%

5%

3%

55%

31%

10%

21%

7%

0%

10%

14%

0%

0%

38%

10%

7%

10%

7%

0%

0%

10%

0%

0%

32%

16%

24%

4%

16%

24%

12%

0%

16%

16%

16%

4%

12%

0%

0%

20%

4%

0%

12%

8%

33%

33%

17%

33%

17%

0%

0%

17%

0%

0%

17%

17%

0%

17%

17%

0%

0%

17%

0%

0%

Base: All Physicians. A1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?

List of suspected

diseases

*Low base**Very low basePlease double click on the icon for the full list: Total ToM

16

Base: All Physicians. A2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?A3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**

I treat myself / I refer to another specialty

Base of physicians who say; “I refer to another specialty”

31 15* 13* 3**

Pediatric Metabolism

Pediatric Rheumatology

Pediatric Endocrinology

Pediatric Gastroenterology

Genetics

48% 52% 48% 52% 48% 52% 50% 50%


Case 1 - Referral


I treat myself

I refer to another specialty

45%

42%

6%

3%

3%

40%

53%

0%

0%

7%

54%

31%

8%

8%

0%

33%

33%

33%

0%

0%

17Base: All Physicians. A3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



Genetics



Pediatric Neurology


Case 1 - Referral Index


53%

30%

8%

4%

2%

2%

48%

34%

15%

0%

0%

3%

61%

26%

0%

6%

6%

0%

48%

26%

0%

26%

0%

0%

18Base: All Physicians. A4. Bu hastaya hangi testleri uygularsınız?


Base 60 29* 25* 6**

Thyroid Tests

Biochemical Tests

Blood Tests

Urine Tests

Celiac Tests

I refer the patient w/o any test

Bone Age Test

IGF-1 Test

Growth hormone / Somatomedin Test

IGF-BP3 Test

Cariotype Analysis

Sedimentation

Hemogram


Case 1 - Tests to RunInstitution Breakdown

42%

40%

33%

27%

25%

25%

23%

22%

17%

10%

8%

8%

8%

41%

48%

45%

34%

28%

17%

34%

21%

14%

7%

17%

10%

14%

48%

36%

20%

8%

24%

36%

16%

28%

16%

16%

0%

8%

4%

17%

17%

33%

67%

17%

17%

0%

0%

33%

0%

0%

0%

0%

Please double click on the icon for the full list:

List of Tests to

Run

19

Case 1 - Additional Information

• Celiac Disease, intestinal parasites, Helicobacter Pylori and Cystic Fibrosis are negative• Recent ultrasonography showed mild growth in the liver without any structural anomaly. • Left hand/wrist plain radiograph: normal bone age, metacarpal bones proximally tapered,

slightly shortened and thickened, among ordinary findings.

Physical Examination History


Base 60 29* 25* 6**

Yes/No

Base of physicians who say “Yes” 9** 7** 2** 0**



15% 85% 24% 76% 8% 92% 0%100%

Base: All Physicians. B0. Son 12 ay içerisinde bu profile uygun bir hasta gördünüz mü? B0x. Son 12 ay içerisinde bu profile uygun kaç hasta gördüğünüzü söyler misiniz?



20


Base 60 29* 25* 6**


Metabolic Diseases

Skeleton / Bone Dysplasia

Turner Syndrome

I don't know




Celiac Disease


Hormonal Disorder

Case 1 - Suspected Diseases (After Additional Information)


22%

18%

18%

17%

7%

7%

5%

5%

5%

5%

5%

18%

15%

15%

15%

7%

7%

0%

3%

3%

2%

2%

Base: All Physicians. B1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?

*Low base**Very low basePlease double click on the icon for the full list:

10%

14%

21%

31%

7%

0%

10%

7%

7%

7%

3%

10%

10%

21%

28%

7%

0%

0%

3%

7%

3%

0%

36%

28%

20%

0%

8%

16%

0%

0%

4%

0%

0%

28%

24%

12%

0%

8%

16%

0%

0%

0%

0%

0%

17%

0%

0%

17%

0%

0%

0%

17%

0%

17%

33%

17%

0%

0%

17%

0%

0%

0%

17%

0%

0%

17%

List of suspected

diseases

Total ToM

21

Total

Base 60





Metabolic Diseases


Celiac Disease

Turner Syndrome

DSD (Disorder Of Sex Development)

Constitutional Short Stature Due To Familial Hypertrophyse

Case 1 - Suspected Diseases (Before/After)

Answers BeforeAdditional Information

43%

25%

17%

15%

12%

10%

10%

8%

7%

7%

27%

8%

8%

7%

5%

8%

2%

7%

5%

3%

Base: All Physicians. A1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?B1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?

Total

Base 60


Metabolic Diseases

Skeleton / Bone Dysplasia

Turner Syndrome

I don't know




Celiac Disease


Hormonal Disorder

22%

18%

18%

17%

7%

7%

5%

5%

5%

5%

5%

18%

15%

15%

15%

7%

7%

0%

3%

3%

2%

2%

Answers AfterAdditional Information

Total ToM

22

Base: All Physicians. B2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?B3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



40 19* 18* 3**




Genetics

33% 67% 34% 66% 28% 72% 50% 50%


Case 1 - Referral (After Additional Information)


I treat myself


48%

43%

5%

5%

37%

58%

0%

5%

67%

28%

6%

0%

0%

33%

33%

33%

23

Base: All Physicians. A2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?A3. Bu hastayı hangi uzmanlığa sevk edersiniz?

Total

Base 60



31





Genetics

48% 52%


Case 1 - Referral (Before/After) I treat myself


45%

42%

6%

3%

3%

Total

Base 60



40




Genetics

33% 67%

48%

43%

5%

5%



24Base: All Physicians. B3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



Genetics



Pediatric Neurology

Pediatric Allergy


Case 1 - Referral Index (After Additional Information)


42%

32%

10%

7%

5%

3%

1%

35%

40%

15%

1%

6%

2%

0%

56%

24%

3%

12%

2%

3%

0%

27%

16%

16%

16%

11%

0%

14%

25

Base: All Physicians. A3. Bu hastayı hangi uzmanlığa sevk edersiniz?B3. Bu hastayı hangi uzmanlığa sevk edersiniz?

Total

Base 60



Genetics



Pediatric Neurology

Case 1 - Referral Index (Before/After)


53%

30%

8%

4%

2%

2%

Total

Base 60



Genetics



Pediatric Neurology

Pediatric Allergy

42%

32%

10%

7%

5%

3%

1%


26Base: All Physicians. B4. Bu hastaya hangi testleri uygularsınız?


Case 1 - Tests to Run (After Additional Information)

Institution Breakdown Total R&T Hospital University Hospital Private Hospital

Base 60 29* 25* 6**


Cariotype Analysis

Genetic Tests

Blood Tests

Urine Tests

Bone/Skeletal Survey

Enzyme Level Tests


Metabolic Survey

IGF-1 Test

Ca Parameters / Calcium Test

40%

15%

13%

12%

12%

8%

7%

5%

5%

5%

5%


31%

28%

14%

10%

10%

7%

3%

3%

7%

7%

7%

56%

0%

12%

8%

4%

12%

8%

0%

0%

4%

4%

17%

17%

17%

33%

50%

0%

17%

33%

17%

0%

0%

List of Tests to

Run

27

Base: All Physicians. A4. Bu hastaya hangi testleri uygularsınız?B4. Bu hastaya hangi testleri uygularsınız?

Case 1 - Tests to Run (Before/After)

Total

Base 60


Cariotype Analysis

Genetic Tests

Blood Tests

Urine Tests

Bone/Skeletal Survey

Enzyme Level Tests


Metabolic Survey

IGF-1 Test

Ca Parameters / Calcium Test

40%

15%

13%

12%

12%

8%

7%

5%

5%

5%

5%

Total

Base 60

Thyroid Tests

Biochemical Tests

Blood Tests

Urine Tests

Celiac Tests


Bone Age Test

IGF-1 Test


IGF-BP3 Test

Cariotype Analysis

Sedimentation

Hemogram

42%

40%

33%

27%

25%

25%

23%

22%

17%

10%

8%

8%

8%



28Base: All Physicians. E1. VAKA I’in aşağıda / göstereceğim hastalıklardan hangileri olabileceğini 5’li skala üzerinden söyler misiniz?


Case 1 - Possible Diseases


Base 60 29* 25* 6**

Myopathy

Other

Rheumatoid Arthritis

Polymyositis

Pompe Disease

Arthrogryposis

Lysosomal Strorage Disease

Collagen Tissue Disease


Metabolic Disease

62%

60%

58%

57%

57%

50%

45%

37%

25%

22%

25%

0%

25%

23%

28%

22%

28%

48%

35%

28%

5%

20%

12%

7%

0%

13%

17%

8%

37%

43%

76%

50%

59%

66%

59%

55%

62%

31%

31%

24%

14%

0%

24%

17%

28%

17%

14%

52%

31%

28%

3%

50%

10%

7%

0%

14%

17%

7%

34%

41%

48%

100%

48%

44%

52%

44%

24%

40%

16%

16%

40%

0%

32%

36%

32%

28%

44%

48%

36%

36%

0%

0%

16%

8%

0%

12%

20%

8%

44%

40%

50%

0%

100%

67%

67%

50%

50%

50%

33%

33%

17%

0%

0%

17%

17%

33%

33%

50%

0%

33%

0%

0%

0%

17%

0%

17%

17%

67%

T2B MB B2B

29Base: All Physicians. E2. Hangi semptomlardan dolayı Vaka I’in MPS I (Mukopolisakaridoz I) hastalığı olmasından şüphelenirsiniz?

Case 1 - Symptoms


Base 22* 10** 11** 1**

Short Stature

Joint Contractures

I don't know

Stomachache

Bone indications / Deformations

Hirsutism

Radiological Findinds

Growth Failure

Hernia / Inguinal Hernia

Hepatomegaly

Snoring

Metabolic Symptoms

Upper Respiratory Infection

Lung Diseases Symptoms

Corneal Haze

Mental Retardation

Myalgia

45%

41%

23%

18%

14%

14%

14%

9%

9%

9%

9%

9%

5%

5%

5%

5%

5%

50%

30%

30%

30%

30%

10%

20%

0%

0%

0%

10%

20%

0%

0%

0%

0%

0%

36%

45%

18%

9%

0%

9%

9%

18%

9%

18%

0%

0%

9%

9%

9%

9%

9%

100%

100%

0%

0%

0%

100%

0%

0%

100%

0%

100%

0%

0%

0%

0%

0%

0%

30

Case 2

Clinical Picture Medical Record

• 15 years old male• His height is at the 25th percentile for his

age and gender but it is below the potential genetic target (mother’s height is at 75th percentile and fathers height is at 97th percentile)

• Normal pubertal maturation• Normal neurological evaluation• Mild scoliosis on the lumbar spine

• Recurrent abdominal pain and continuous diarrhea-constipation pattern from age 5 (diagnosis of irritable bowel syndrome)

• Lactose intolerance• Adenotonsillectomy operation due to otitis media and recurrent upper respiratory

tract infections at the age of 10• Recurrent urinary tract infections during the first 3 years of life• Bilateral joint stiffness with slow progression in the upper and lower extremities

starting from age 4 with the limitation of motion range• Uses inhaled steroids for asthma• When the family history is considered, his grandfather has a history of rheumatoid

arthritis

Base: All Physicians. C0. Son 12 ay içerisinde bu profile uygun bir hasta gördünüz mü? C0x. Son 12 ay içerisinde bu profile uygun kaç hasta gördüğünüzü söyler misiniz?


Base 60 29* 25* 6**

Yes/No




10% 90% 10% 90% 4% 96% 33% 67%



31


Base 60 29* 25* 6**


Metabolic Diseases


I don't know

Celiac Disease

Lysosomal Storage Disease

Genetic Disorders

Immunologic Diseases

Scoliosis

Case 2 - Suspected Diseases at First


42%

18%

15%

12%

10%

7%

7%

7%

7%

38%

10%

12%

10%

8%

0%

7%

3%

0%

Base: All Physicians. C1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?

List of suspected

diseases

*Low base**Very low basePlease double click on the icon for the full list:

48%

24%

10%

10%

10%

10%

7%

10%

7%

45%

7%

10%

10%

10%

0%

7%

7%

0%

36%

16%

24%

12%

8%

0%

0%

4%

0%

32%

16%

16%

8%

8%

0%

0%

0%

0%

33%

0%

0%

17%

17%

17%

33%

0%

33%

33%

0%

0%

17%

0%

0%

33%

0%

0%

Total ToM

32

Base: All Physicians. C2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?C3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



52 26* 22* 4**




Genetics


Other

13% 87% 10% 90% 12% 88% 33% 67%


Case 2 - Referral


I treat myself


58%

27%

6%

6%

2%

2%

62%

23%

8%

8%

0%

0%

55%

36%

5%

5%

0%

0%

50%

0%

0%

0%

25%

25%

33Base: All Physicians. C3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



Genetics



Other

Pediatric Neurology


Case 2 - Referral (Index)


41%

34%

10%

7%

4%

3%

2%

41%

35%

9%

10%

3%

2%

0%

43%

31%

12%

5%

2%

2%

4%

37%

35%

0%

0%

14%

14%

0%

34Base: All Physicians. C4. Bu hastaya hangi testleri uygularsınız?


Base 60 29* 25* 6**


Blood Tests

Urine Tests

Biochemical Tests

Celiac Tests

Metabolic Analysis

Urinary GAG Testing

Genetic Tests

Bone Age Test

Sedimentation



Case 2 - Tests to Run


55%

18%

12%

10%

10%

10%

8%

7%

7%

7%

5%


55%

17%

14%

7%

10%

17%

17%

3%

7%

3%

7%

64%

12%

4%

16%

8%

0%

0%

8%

8%

12%

0%

17%

50%

33%

0%

17%

17%

0%

17%

0%

0%

17%

List of Tests to

Run

35

Case 2 - Additional Information

• Physical Examination shows;– Mild corneal opacity– Cardiac souffle

• Previous hematological examinations show;– Inflammatory indicators; negative– Celiac Disease indicators; negative

Physical Examination History


Base 60 29* 25* 6**

Yes/No




7% 93% 7% 93% 4% 96% 17% 83%

Base: All Physicians. D0. Son 12 ay içerisinde bu profile uygun bir hasta gördünüz mü? D0x. Son 12 ay içerisinde bu profile uygun kaç hasta gördüğünüzü söyler misiniz?



36


Base 60 29* 25* 6**

Metabolic Diseases




I don't know

Diabetes

Genetic Disorders

Celiac Disease

Cardiological Diseases

Scoliosis


Skeletal / Bone Dysplasia

FMF Disease

Hormonal Disorder

Systemic Lupus Erythematosus (SLE)

Case 2 - Suspected Diseases (After Additional Information)


28%

25%

22%

18%

13%

5%

3%

3%

3%

3%

2%

2%

2%

2%

2%

25%

18%

15%

17%

13%

3%

3%

0%

2%

0%

0%

0%

2%

0%

2%

Base: All Physicians. D1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?


17%

17%

31%

17%

14%

3%

3%

3%

7%

7%

3%

0%

0%

0%

3%

17%

10%

28%

17%

14%

3%

3%

0%

3%

0%

0%

0%

0%

0%

3%

44%

36%

12%

20%

12%

0%

0%

0%

0%

0%

0%

4%

4%

0%

0%

17%

17%

17%

17%

17%

33%

17%

17%

0%

0%

0%

0%

0%

17%

0%

17%

0%

17%

17%

17%

17%

17%

0%

0%

0%

0%

0%

0%

0%

0%

Total ToM

37

Total

Base 60

Metabolic Diseases




I don't know

Diabetes

Genetic Disorders

Celiac Disease

Cardiological Diseases

Scoliosis


Skeletal / Bone Dysplasia

FMF Disease

Hormonal Disorder

Systemic Lupus Erythematosus (SLE)

Case 2 - Suspected Diseases (Before/After)

28%

25%

22%

18%

13%

5%

3%

3%

3%

3%

2%

2%

2%

2%

2%

25%

18%

15%

17%

13%

3%

3%

0%

2%

0%

0%

0%

2%

0%

2%

Base: All Physicians. C1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?D1. Profili ve klinik muayenesi bu şekilde olan bir hastada hangi hastalıklardan şüphelenirsiniz?

Total

Base 60


Metabolic Diseases


I don't know

Celiac Disease


Genetic Disorders


Scoliosis

42%

18%

15%

12%

10%

7%

7%

7%

7%

38%

10%

12%

10%

8%

0%

7%

3%

0%



Total ToM

38

Base: All Physicians. D2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?D3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



52 26* 22* 4**




Genetics


Pediatric Neurology

13% 87% 10% 90% 12% 88% 33% 67%


Case 2 - Referral (After Additional Information)


I treat myself


65%

23%

4%

4%

2%

2%

65%

23%

4%

4%

4%

0%

68%

23%

5%

5%

0%

0%

50%

25%

0%

0%

0%

25%

39

Base: All Physicians. C2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?C3. Bu hastayı hangi uzmanlığa sevk edersiniz? D2. Bu hastayı kendiniz mi tedavi edersiniz ya da bir başka uzmanlığa mı sevk edersiniz?D3. Bu hastayı hangi uzmanlığa sevk edersiniz?

Total

Base 60



52




Genetics


Pediatric Neurology

13% 87%

Case 2 - Referral (Before/After) I treat myself


65%

23%

4%

4%

2%

2%

Total

Base 60



52




Genetics


Other

13% 87%

58%

27%

6%

6%

2%

2%



40Base: All Physicians. D3. Bu hastayı hangi uzmanlığa sevk edersiniz?


Base 60 29* 25* 6**



Genetics

Other


Pediatric Neurology



Case 2 - Referral Index (After Additional Information)


51%

25%

10%

5%

4%

3%

1%

47%

29%

13%

6%

3%

0%

3%

60%

21%

5%

3%

7%

5%

0%

36%

18%

12%

15%

0%

18%

0%

41

Base: All Physicians. C3. Bu hastayı hangi uzmanlığa sevk edersiniz?D3. Bu hastayı hangi uzmanlığa sevk edersiniz?

Total

Base 60



Genetics



Other

Pediatric Neurology

Case 2 - Referral Index (Before/After)

41%

34%

10%

7%

4%

3%

2%

Total

Base 60



Genetics

Other


Pediatric Neurology


51%

25%

10%

5%

4%

3%

1%



42Base: All Physicians. D4. Bu hastaya hangi testleri uygularsınız?


Case 2 - Tests to Run (After Additional Information)

Institution Breakdown Total R&T Hospital University Hospital Private Hospital

Base 60 29* 25* 6**


Urinary GAG Testing

Blood Tests

Urine Tests

Genetic Tests

Boichemical Tests


Enzyme Level Tests

Bone Age Test

Cariotype Analysis

Hemoglobin

40%

15%

13%

12%

12%

8%

7%

5%

5%

5%

5%


55%

24%

7%

7%

7%

7%

3%

0%

7%

3%

3%

68%

0%

8%

8%

8%

4%

4%

8%

0%

0%

4%

50%

17%

33%

33%

0%

0%

0%

0%

0%

17%

0%

List of Tests to

Run

43

Base: All Physicians. C4. Bu hastaya hangi testleri uygularsınız?D4. Bu hastaya hangi testleri uygularsınız?

Case 2 - Tests to Run (Before/After)

Total

Base 60


Urinary GAG Testing

Blood Tests

Urine Tests

Genetic Tests

Boichemical Tests


Enzyme Level Tests

Bone Age Test

Cariotype Analysis

Hemoglobin

40%

15%

13%

12%

12%

8%

7%

5%

5%

5%

5%

Total

Base 60


Blood Tests

Urine Tests

Biochemical Tests

Celiac Tests

Metabolic Analysis

Urinary GAG Testing

Genetic Tests

Bone Age Test

Sedimentation


55%

18%

12%

10%

10%

10%

8%

7%

7%

7%

5%

44Base: All Physicians. E3. VAKA II’nin aşağıda / göstereceğim hastalıklardan hangileri olabileceğini 5’li skala üzerinden söyler misiniz?


Case 2 - Possible Diseases


Base 60 29* 25* 6**

Arthrogryposis

Polymyositis

Pompe Disease

Myopathy

Other

Collagen Tissue Disease

Lysosomal Strorage Disease

Rheumatoid Arthritis


Metabolic Disease

55%

55%

55%

53%

50%

38%

37%

37%

27%

17%

27%

27%

22%

23%

0%

32%

22%

32%

32%

30%

5%

2%

3%

10%

13%

18%

30%

25%

33%

40%

59%

52%

48%

52%

40%

38%

41%

41%

31%

17%

24%

28%

17%

17%

0%

31%

10%

24%

21%

21%

3%

0%

3%

10%

0%

14%

31%

24%

34%

41%

56%

50%

28%

52%

56%

52%

32%

36%

20%

16%

32%

0%

48%

32%

32%

32%

36%

40%

40%

40%

4%

50%

20%

4%

0%

4%

24%

16%

36%

36%

50%

100%

50%

83%

83%

50%

33%

50%

33%

17%

17%

0%

0%

0%

17%

17%

0%

50%

33%

33%

50%

0%

17%

17%

50%

50%

17%

50%

T2B MB B2B

45Base: All Physicians. E4. Hangi semptomlardan dolayı Vaka II’nin MPS I (Mukopolisakaridoz I) hastalığı olmasından şüphelenirsiniz?

Case 2 - Symptoms


Base 20* 10** 9** 1**

Short Stature

Corneal Opacity

I don't know

Joint Contractures

Scoliosis

Stomachache

Bone indications / Deformations

Growth Failure

Genetic Disorders

Upper Respiratory Infection

Corneal Haze

Cataract

Hernia / Inguinal Hernia

Hepatomegaly

Lordosis

Physical Problems

Mental Retardation

35%

35%

30%

25%

20%

15%

15%

10%

10%

10%

10%

5%

5%

5%

5%

5%

5%

20%

20%

50%

10%

30%

30%

10%

10%

20%

0%

0%

10%

0%

0%

0%

0%

0%

56%

56%

11%

33%

11%

0%

22%

11%

0%

22%

11%

0%

11%

0%

11%

0%

11%

0%

0%

0%

100%

0%

0%

0%

0%

0%

0%

100%

0%

0%

100%

0%

100%

0%

47


Base 60 29* 25* 6**

5% 10%0% 0%

52% 45% 64%

33%

23% 31% 12%

33%

17%14%

20%

17%

3% 0% 4%17%

Very well informed

Informed

Have enough information

Have some information

I do not have any information

Information Level of MPS1 Disease

% of physicians

Base: All Physicians. E5. MPS I (Mukopolisakaridoz I) hastalığı hakkındaki bilgi seviyenizi 1 ile 5 arasındaki skalada değerlendirebilir misiniz?



48

Information Level of MPS1 Disease (Top 2)



Base 60 29* 25* 6**

57% 55%64%

33%

23% 31% 12%

33%

20% 14%24%

33%TOP 2(Informed & Very wellinformed)

MID(Have enough information)

BOT 2(I do not have any information& Have some information)



49


Base 60 29* 25* 6**

3% 7%0% 0%

57% 48% 68%

50%

27% 38% 16%

17%

10%7%

12%

17%

3% 4%17%

Very well informed

Informed




Information Level of MPS1 Treatment Methods

% of physicians

Base: All Physicians. E6. MPS I (Mukopolisakaridoz I) hastalığının TEDAVİSİ hakkındaki bilgi seviyenizi 1 ile 5 arasındaki skalada değerlendirebilir misiniz?



50

Information Level of MPS1 Treatment Methods (Top 2)

Base: All Physicians. E6. MPS I (Mukopolisakaridoz I) hastalığının TEDAVİSİ hakkındaki bilgi seviyenizi 1 ile 5 arasındaki skalada değerlendirebilir misiniz?


Base 60 29* 25* 6**

60% 55%68%

50%

27% 38% 16%

17%

13% 7%16%

33%TOP 2(Informed & Very wellinformed)


BOT 2(I do not have any information& Have some information)



51Base: All Physicians. E7. MPS I (Mukopolisakaridoz I) hastalığı tanısı için uygulanan testlerden bildiklerinizi söyler misiniz? Başka hangi testleri biliyorsunuz?


Known MPS 1 TestsInstitution Breakdown Total R&T Hospital University Hospital Private Hospital

Base 60 29* 25* 6**

Genetic Tests

Urine Tests

I don't know

Blood Tests

Enzyme Level Tests

Metabolic Analysis

Skin Biopsy

MPS in Urine

Boichemical Tests

MPS Test

IGF-1 Test

43%

25%

23%

23%

17%

10%

5%

5%

3%

3%

3%

22%

20%

23%

7%

7%

3%

2%

3%

2%

0%

0%


45%

17%

24%

10%

10%

14%

7%

7%

0%

3%

0%

34%

10%

24%

3%

7%

3%

3%

3%

0%

0%

0%

36%

28%

28%

24%

24%

4%

0%

4%

4%

4%

4%

12%

24%

28%

8%

8%

0%

0%

4%

4%

0%

0%

67%

50%

0%

83%

17%

17%

17%

0%

17%

0%

17%

0%

50%

0%

17%

0%

17%

0%

0%

0%

0%

0%

List of Known

Tests

Total ToM

52Base: All Physicians. E8. MPS I (Mukopolisakaridoz I) hastalığı için uygulanan tedavilerden bildiklerinizi söyler misiniz?


Known MPS 1 TestsInstitution Breakdown Total R&T Hospital University Hospital Private Hospital

Base 60 29* 25* 6**

Enzyme Level Tests

I don't know

Bone marrow transplantation test

Physiotherapy

Medical treatment

Kidney Function Tests

Tandem MS tests

MPS Test

Tracheostomy

Heart

Oxygen support

Skeletal Survey

Stem Cell Transplantation

Endocrine Test

58%

37%

7%

5%

3%

2%

2%

2%

2%

2%

2%

2%

2%

2%

55%

32%

0%

5%

2%

0%

2%

2%

2%

0%

2%

0%

0%

0%

58%

37%

7%

5%

3%

2%

2%

2%

2%

2%

2%

2%

2%

2%

55%

34%

0%

3%

3%

0%

0%

0%

0%

0%

3%

0%

0%

0%

58%

37%

7%

5%

3%

2%

2%

2%

2%

2%

2%

2%

2%

2%

56%

36%

0%

0%

0%

0%

4%

4%

0%

0%

0%

0%

0%

0%

58%

37%

7%

5%

3%

2%

2%

2%

2%

2%

2%

2%

2%

2%

50%

0%

0%

33%

0%

0%

0%

0%

17%

0%

0%

0%

0%

0%

53

MPS1 Incidence and Tests Ran

Base: All Physicians. E9. Şimdiye kadar hiçbir hastanıza MPS I (Mukopolisakaridoz I) tanısı koydunuz mu?E9a. Son 2 yıl içerisinde kaç hastaya MPS I (Mukopolisakaridoz I) tanısı koydunuz?E10. MPS I (Mukopolisakaridoz I) tanısı koyduğunuz hastalara hangi testleri uyguladığınızı söyler misiniz?


Base 60 29* 25* 6**

Yes/No




Enzyme Level

Genetic Tests

Blood Tests

Urine Tests

Liver Tests

MPS in Urine

Glucosamine in Urine

13% 87% 10% 90% 12% 88% 33% 67%



38%

25%

25%

25%

13%

13%

13%

67%

0%

0%

0%

33%

0%

0%

33%

33%

0%

0%

0%

33%

33%

0%

50%

100%

100%

0%

0%

0%

54

MPS1 Incidence - Specialties

Base: All Physicians. E11. MPS I (Mukopolisakaridoz I) tanısı koyduğunuz hastaların tedavisinde hangi uzmanlıklarla birlikte çalıştınız?


Base 8** 3** 3** 2**

Pediatric Metabolism 88% 100% 67% 100%

Pediatric Neurology 25% 0% 33% 50%

Endocrinology 13% 0% 0% 50%

Genetics 63% 0% 100% 100%



56

% Total R&T Hospital University Hospital Private Hospital

Base 60 29* 25* 6**

Yes (I would like to be informed about the disease)

No ( I wouldn’t like to be informed about the disease)

Information Demand For MPS1 Disease

43

57

Yes

No

59

41

28

72

33

67

Treatment

Diagnostics tests

Sub-types

Symptoms

Prognosis

Follow-up

On which issues…

94%

76%

76%

65%

41%

41%

100%

100%

100%

100%

100%

86%

100%

50%

50%

0%

0%

0%

Base 26* 17* 7** 2**


Base: All Physicians. E12. MPS I (Mukopolisakaridoz I) hastalığı ile ilgili bilgi almak ister misiniz?E13. MPS I (Mukopolisakaridoz I) hastalığı ile ilgili hangi konularda bilgi almak istersiniz?

96%

81%

81%

69%

54%

50%

57

% Total

Base 60



Total ( ) Total ( )

Base 26* 34

12%0%

42% 59%

31% 18%

12% 21%

4% 3%

Very well informed

Informed




Information Level of MPS1 DiseasePhysicians who would like to be informed

% of physicians


*Low base

43

57

YesNo

Yes No

58

Information Level of MPS1 DiseasePhysicians who would like to be informed


% Total

Base 60



Total ( ) Total ( )

Base 26* 34

% of physicians

*Low base

43

57

YesNo

Yes No

54% 59%

31% 18%

15%24%TOP 2

(Informed & Very well informed)


BOT 2(I do not have any information &Have some information)

59Base: All Physicians. E14. Peki bu bilgileri hangi kaynaklardan edinmek istersiniz?E15. Aşağıda bulunan MPS I (Mukopolisakaridoz I) hastalığına dair bilgi kaynaklarını tercih sıranıza göre sıralayabilir misiniz?

Information Resources Preferred


Base 26* 17* 7** 2**

Scientific meetings

Congress

Medical publications/reference books

Websites (associations & organizations)

E-mail

SRs

Websites (pharmaceutial companies)

Webcast

65%

58%

46%

27%

23%

8%

8%

4%

* Low base Numbers show the index of the most preferred information channels

• Physicians are mostly willing to be informed by Scientific Meetings, Congresses and Medical publications/reference books..

65%

53%

47%

35%

24%

6%

12%

6%

71%

71%

43%

0%

29%

0%

0%

0%

50%

50%

50%

50%

0%

50%

0%

0%

1

2

3

4

1

2

3

4

1

2

3

4

1

1

2

2

60

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