Children Patient Case Studies
See how CRYSVITA may help your patients
with XLH
See how CRYSVITA may help your patients
with XLH
Patient growth evolution prior to and after CRYSVITA.
A: At 24.5 months, the patient was in the 1st percentile for height.
B: At enrollment, the patient was in the 5th percentile for height.
C: While on CRYSVITA, the patient was in the 10th percentile for height.
†The reference percentiles on the graph are combined from the two clinical growth charts for boys 2-20 years of age provided by the Centers for Disease Control and Prevention.
Evaluation at academic center
CRYSVITA treatment, 64 weeks
Test (reference rangea unit) | Results (age) | |||
---|---|---|---|---|
Early evaluation (24.5 months) |
Prior to CRYSVITA (31 months) |
CRYSVITA 40 weeks (40 months) |
CRYSVITA 64 weeks (46 months) |
|
Serum phosphorus (4.3-6.8 mg/dL) | 2.9 | 2.9 | 3.7 | 4 |
TmP/GFR (4.0-5.2 mg/dL) | n/a | 2.8 | 3.7 | 4 |
25(OH)D (≥20 ng/mL) | 26 | 24 | 36 | 26 |
ALP (156-369 U/L) | 636 | 459 | 283 | 299 |
Serum calcium (9.2-10.5 mg/dL) | 9.4 | 9.5 | 9.6 | 9.6 |
PTH (10-65 pg/mL) | 56 | 20 | 36 | 23 |
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25(OH)D, 25-hydroxy vitamin D (calcifediol); ALP, alkaline phosphatase; PTH, parathyroid hormone; TmP/GFR, ratio of tubular maximum reabsorption of phosphate to glomerular filtration rate.
aIndicates normal range, age, and sex matched. Note that normal range values may vary depending on reference dataset. The range in this table was provided by the treating physician.
Patient growth evolution prior to and after CRYSVITA.
A: At 26 months, the patient was in the 3rd to 5th percentile for height.
B: At enrollment, the patient was in the 10th percentile for height.
C: While on CRYSVITA, the patient was between the 10th and 15th percentile for height.
†The reference percentiles on the graph are combined from the two clinical growth charts for girls 2-20 years of age provided by the Centers for Disease Control and Prevention.
Evaluation at academic center
CRYSVITA treatment, 106 weeks
Results (age) | ||||||
---|---|---|---|---|---|---|
Test (reference rangea unit) | Early evaluation (24 months) |
Early evaluation (26 months) |
Prior to CRYSVITA (37 months) |
CRYSVITA 40 weeks (47 months) |
CRYSVITA 64 weeks (53 months) |
CRYSVITA 106 weeks (63 months) |
Serum phosphorus (4.3-6.8 mg/dL) | 2.7 | 3.0 | 2.5 | 3.7 | 3.4 | 3.9 |
TmP/GFR (4.0-5.2 mg/dL) | n/a | 2.5 | 2.15 | n/a | 2.79 | 3.91 |
25(OH)D (≥20 ng/mL) | 27 | n/a | 40 | n/a | 30 | n/a |
ALP (156-369 U/L) | 487 | 502 | 528 | 369 | 232 | 295 |
Serum calcium (9.2-10.5 mg/dL) | 9.9 | 9.8 | 9.3 | 9.2 | 8.9 | 9.4 |
PTH (10-65 pg/mL) | 48 | n/a | 33 | 31 | 44 | n/a |
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25(OH)D, 25-hydroxy vitamin D (calcifediol); ALP, alkaline phosphatase; PTH, parathyroid hormone; TmP/GFR, ratio of tubular maximum reabsorption of phosphate to glomerular filtration rate.
aIndicates normal range, age, and sex matched. Note that normal range values may vary depending on reference dataset. The range in this table was provided by the treating physician.
XLH symptoms and associated complications
CRYSVITA treatment, 3.8 years
Patient growth evolution prior to and after CRYSVITA.
A: Prior to 6 years, the patient’s height was at the 3rd to 5th percentile.
B: At enrollment in the CRYSVITA clinical trial, the patient’s height was at the 3rd to 5th percentile.
C: During CRYSVITA treatment, the patient’s height increased to the 10th to 25th percentile.
†The reference percentiles on the graph are combined from the two clinical growth charts for girls 2-20 years of age in Sets 1 and 2 of the clinical charts provided by the Centers for Disease Control and Prevention.
Results (age) | |||||
---|---|---|---|---|---|
Test (reference rangea unit) | Early evaluation (8.8 years) |
Prior to CRYSVITA (8.9 years) |
CRYSVITA 40 weeks (9.6 years) |
CRYSVITA 2 years (11 years) |
CRYSVITA 3.8 years (12 years) |
Serum phosphorus (3.2-6.1 mg/dL) |
2.4 | 1.9 | 3.4 | 3.6 | 3.2 |
TmP/GFR (4.0-5.2 mg/dL) |
n/a | 1.7 | 3.1 | 3.2 | 3.0 |
25(OH)D (20-50 ng/mL) |
n/a | 38 | n/a | 29 | 24 |
ALP (156-369 U/L) |
492 | 490 | 538 | 404 | 300 |
Serum calcium (8.8-10.3 mg/dL) |
9.5 | 9.7 | n/a | 9.7 | 9.4 |
PTH (18-80 pg/mL) |
16 | 38 | 45 | 38 | 70 |
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25(OH)D, 25-hydroxy vitamin D (calcifediol); ALP, alkaline phosphatase; PTH, parathyroid hormone; TmP/GFR, ratio of tubular maximum reabsorption of phosphate to glomerular filtration rate.
aIndicates normal range, age, and sex matched. Note that normal range values may vary depending on reference dataset.
The range in this table was provided by the treating physician.
See how CRYSVITA may help your patients
with XLH
XLH symptoms and associated complications
CRYSVITA treatment
Patient growth evolution.
A: At 7 years, the patient’s height was below the 3rd percentile.
B: At 13 years, the patient’s height continued to be below the 3rd percentile.
C: Final adult height remained below the 3rd percentile.
†The reference percentiles on the graph are combined from the two clinical growth charts for girls 2-20 years of age in Sets 1 and 2 of the clinical charts provided by the Centers for Disease Control and Prevention.
Test (reference rangea unit) |
Results (age) | |||
---|---|---|---|---|
Conventional therapy (12 years) |
Conventional therapy (23 years) |
CRYSVITA (23.5 years) |
CRYSVITA (25 years) |
|
Serum phosphorus (2.5-4.5 mg/dL) |
2.0 | 1.9 | 3.1 | 2.9 |
TmP/GFR (2.5-4.2 mg/dL) |
n/a | 1.5 | 2.7 | n/a |
25(OH)D (20-50 ng/mL) |
n/a | 28 | 25 | n/a |
Serum calcium (8.6-10.2 mg/dL) |
9.7 | 8.9 | 8.7 | 9.3 |
PTH (14-72 pg/mL) |
57 | 49 | n/a | n/a |
25(OH)D, 25-hydroxy vitamin D (calcifediol); PTH, parathyroid hormone; TmP/GFR, ratio of tubular maximum reabsorption of phosphate to glomerular filtration rate.
aIndicates normal range, age, and sex matched. Note that normal range values may vary depending on reference dataset.
The range in this table was provided by the reference laboratory.
*The information for this case study was provided courtesy of Dr. Anthony Portale, Director of the Pediatric Dialysis Program, UCSF Benioff Children’s Hospital, San Francisco. This case study represents a real patient and is intended to be illustrative, not a recommendation for treatment or management. Not all patients will respond the same to CRYSVITA treatment. Results may vary. This case study does not claim to represent typical results.
Integrative medicine evaluation
X-ray evaluation at age 26 (see X-ray 2)
Metabolic clinic follow-up evaluation at age 27
CRYSVITA treatment, 21 weeks
Test (reference rangea unit) | Results (age) | |
---|---|---|
Prior to CRYSVITA (27 years) |
CRYSVITA 21 weeks (27 years) |
|
Serum phosphorus (2.3-4.7 mg/dL) | 1.2 | 3.1 |
1,25 (OH)2 (20-79 ng/mL) | 31.4 | 73 |
25(OH)D (25-80 ng/mL) | 36 | 30 |
BSAP (4.5-16.9 mcg/L) | 21.3 | 19.2 |
PTH (16-88 pg/mL) | 54 | 43 |
Creatinine (0.57-1.11 mg/dL) | - | 0.73 |
1,25 (OH)2, 1,25 25-hydroxy vitamin D; 25(OH)D, 25-hydroxy vitamin D (calcifediol); BSAP, bone-specific alkaline phosphatase, also known as BAP; PTH, parathyroid hormone.
aIndicates normal range, age, and sex matched. Note that normal range values may vary depending on reference dataset. The range in this table was provided by the treating physician.
*The information for this case study was provided courtesy of Dr. Kathyryn McCrystal Dahir, Professor of Medicine, Department of Endocrinology, Vanderbilt University Medical Center, Nashville. This case study represents a real patient and is intended to be illustrative, not a recommendation for treatment or management. Not all patients will respond the same to CRYSVITA treatment. Results may vary. This case study does not claim to represent typical results.
CRYSVITA treatment, 35 weeks
Test (reference rangea unit) | Results (age) | |
---|---|---|
Early evalution (35 years) |
CRYSVITA 35 weeks (37 years) |
|
Serum phosphorus (2.3-4.7 mg/dL) | 1.6 | 2.6 |
1,25 (OH)2 (20-80 ng/mL) | 40.3 | - |
25(OH)D (25-80 ng/mL) | 35 | 60.6 |
BSAP (4.5-16.9 mcg/L) | 25 | 19.1 |
PTH (16-88 pg/mL) | 55 | 52 |
Creatinine (0.57-1.11 mg/dL) | 0.74 | 0.70 |
1,25 (OH)2, 1,25 25-hydroxy vitamin D; 25(OH)D, 25-hydroxy vitamin D (calcifediol); BSAP, bone-specific alkaline phosphatase, also known as BAP; PTH, parathyroid hormone.
aIndicates normal range, age, and sex matched. Note that normal range values may vary depending on reference dataset. The range in this table was provided by the treating physician.
*The information for this case study was provided courtesy of Dr. Kathyryn McCrystal Dahir, Professor of Medicine, Department of Endocrinology, Vanderbilt University Medical Center, Nashville. This case study represents a real patient and is intended to be illustrative, not a recommendation for treatment or management. Not all patients will respond the same to CRYSVITA treatment. Results may vary. This case study does not claim to represent typical results.
Test (reference rangea unit) | Results (age) | |||
---|---|---|---|---|
Conventional therapy (12 years) |
Conventional therapy (23 years) |
CRYSVITA (23.5 years) |
CRYSVITA (25 years) |
|
Serum phosphorus (2.5-4.5 mg/dL) |
2.0 | 1.9 | 3.1 | 2.9 |
TmP/GFR (2.5-4.2 mg/dL) |
n/a | 1.5 | 2.7 | n/a |
25(OH)D (20-50 ng/mL) |
n/a | 28 | 25 | n/a |
Serum calcium (8.6-10.2 mg/dL) |
9.7 | 8.9 | 8.7 | 9.3 |
PTH (14-72 pg/mL) |
57 | 49 | n/a | n/a |
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At 12 years old: coxa vara, genu varum, femoral and mild tibial bowing, rachitic changes at knee and wrist (arrows).
Test (reference rangea unit) | Results (age) | |
---|---|---|
Prior to CRYSVITA (27 years) |
CRYSVITA 21 weeks (27 years) |
|
Serum phosphorus (2.3-4.7 mg/dL) | 1.2 | 3.1 |
1,25 (OH)2 (20-79 ng/mL) | 31.4 | 73 |
25(OH)D (25-80 ng/mL) | 36 | 30 |
BSAP (4.5-16.9 mcg/L) | 21.3 | 19.2 |
PTH (16-88 pg/mL) | 54 | 43 |
Creatinine (0.57-1.11 mg/dL) | - | 0.73 |
X-Ray 2: Enthesopathy
At 26 years old: bilateral calcaneal enthesophytes; previously broken left Achilles enthesophyte; small bilateral Haglund bumps with mild bilateral retrocalcaneal bursitis.
At 27 years old: significant discomfort in the anteromedial aspect of the right knee with use. Proximal tibial osteotomy performed when patient was a teenager.
Test (reference rangea unit) | Results (age) | |
---|---|---|
Early evalution (35 years) |
CRYSVITA 35 weeks (37 years) |
|
Serum phosphorus (2.3-4.7 mg/dL) | 1.6 | 2.6 |
1,25 (OH)2 (20-80 ng/mL) | 40.3 | - |
25(OH)D (25-80 ng/mL) | 35 | 60.6 |
BSAP (4.5-16.9 mcg/L) | 25 | 19.1 |
PTH (16-88 pg/mL) | 55 | 52 |
Creatinine (0.57-1.11 mg/dL) | 0.74 | 0.70 |
Test (reference rangea unit) | Results (age) | |||
---|---|---|---|---|
Early evaluation (24.5 months) |
Prior to CRYSVITA (31 months) |
CRYSVITA 40 weeks (40 months) |
CRYSVITA 64 weeks (46 months) |
|
Serum phosphorus (4.3-6.8 mg/dL) | 2.9 | 2.9 | 3.7 | 4 |
TmP/GFR (4.0-5.2 mg/dL) | n/a | 2.8 | 3.7 | 4 |
25(OH)D (≥20 ng/mL) | 26 | 24 | 36 | 26 |
ALP (156-369 U/L) | 636 | 459 | 283 | 299 |
Serum calcium (9.2-10.5 mg/dL) | 9.4 | 9.5 | 9.6 | 9.6 |
PTH (10-65 pg/mL) | 56 | 20 | 36 | 23 |
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X-RAYS: Evolution of Rickets and Lower Limb Deformity
Prior to CRYSVITA
(29 months old)
CRYSVITA 40 weeks
(40 months old)
CRYSVITA 64 weeks
(46 months old)
Cupping and fraying of growth plates, marked bowing.
Prior to CRYSVITA
(29 months old)
CRYSVITA 40 weeks
(40 months old)
CRYSVITA 64 weeks
(46 months old)
Resolved cupping and fraying of metaphysis of radius and ulna; improved fraying of femoral metaphysis; decreased widening of femoral growth plate.
Results (age) | ||||||
---|---|---|---|---|---|---|
Test (reference rangea unit) | Early evaluation (24 months) |
Early evaluation (26 months) |
Prior to CRYSVITA (37 months) |
CRYSVITA 40 weeks (47 months) |
CRYSVITA 64 weeks (53 months) |
CRYSVITA 106 weeks (63 months) |
Serum phosphorus (4.3-6.8 mg/dL) | 2.7 | 3.0 | 2.5 | 3.7 | 3.4 | 3.9 |
TmP/GFR (4.0-5.2 mg/dL) | n/a | 2.5 | 2.15 | n/a | 2.79 | 3.91 |
25(OH)D (≥20 ng/mL) | 27 | n/a | 40 | n/a | 30 | n/a |
ALP (156-369 U/L) | 487 | 502 | 528 | 369 | 232 | 295 |
Serum calcium (9.2-10.5 mg/dL) | 9.9 | 9.8 | 9.3 | 9.2 | 8.9 | 9.4 |
PTH (10-65 pg/mL) | 48 | n/a | 33 | 31 | 44 | n/a |
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X-rays: Evolution of Rickets and Lower Limb Deformity
Early evaluation
(26 months old)
Prior to CRYSVITA
(37 months old)
CRYSVITA 40 weeks
(47 months old)
CRYSVITA 64 weeks
(53 months old)
CRYSVITA 106 weeks
(63 months old)
Results (age) | |||||
---|---|---|---|---|---|
Test (reference rangea unit) | Early evaluation (8.8 years) |
Prior to CRYSVITA (8.9 years) |
CRYSVITA 40 weeks (9.6 years) |
CRYSVITA 2 years (11 years) |
CRYSVITA 3.8 years (12 years) |
Serum phosphorus (3.2-6.1 mg/dL) |
2.4 | 1.9 | 3.4 | 3.6 | 3.2 |
TmP/GFR (4.0-5.2 mg/dL) |
n/a | 1.7 | 3.1 | 3.2 | 3.0 |
25(OH)D (20-50 ng/mL) |
n/a | 38 | n/a | 29 | 24 |
ALP (156-369 U/L) |
492 | 490 | 538 | 404 | 300 |
Serum calcium (8.8-10.3 mg/dL) |
9.5 | 9.7 | n/a | 9.7 | 9.4 |
PTH (18-80 pg/mL) |
16 | 38 | 45 | 38 | 70 |
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X-rays: Evolution of Rickets and Lower Limb Deformity
Prior to CRYSVITA
(8 years old)
Active rickets at knee.
CRYSVITA 3.8 years
(12 years old)
Mild widening of physis of distal femur.
Prior to CRYSVITA
(8 years old)
Femoral bowing, coxa vara.
CRYSVITA 3.8 years
(12 years old)
Persistent femoral bowing.