PMID- 8065868
OWN - NLM
STAT- MEDLINE
DCOM- 19940922
LR  - 20181130
IS  - 0031-4005 (Print)
IS  - 0031-4005 (Linking)
VI  - 94
IP  - 3
DP  - 1994 Sep
TI  - Aminohydroxy propylidene bisphosphonate (APD) treatment improves the clinical 
      skeletal manifestations of Gaucher's disease.
PG  - 385-9
AB  - OBJECTIVE: To evaluate the long-term effects and safety of aminohydroxy 
      propylidene bisphosphonate (APD) treatment on the frequency and severity of the 
      clinical skeletal manifestations of Gaucher's disease. METHODOLOGY: Five 
      adolescents who suffered from recurrent bone crisis episodes and atraumatic bone 
      fractures due to Gaucher's disease were treated with APD for 14 to 83 months. 
      RESULTS: During the 6 years before treatment, the patients suffered from 6 to 17 
      bone crisis episodes, or 1 to 2.8 episodes per patient per year. Three patients 
      were free from bone crisis episodes during 14 to 32 months of ADP treatment, 
      while two patients had two such episodes during 60 and 83 months of APD treatment 
      (these represent a decrease in bone crisis episodes from 1 and 2.8 per year to 
      0.4 and 0.3 per year, respectively). Although four patients suffered from 1 to 3 
      atraumatic bone fractures during the 6 years preceding treatment (a total of 10 
      fractures), only one patient sustained a fracture on APD treatment (total of 219 
      months of treatment). Using APD was not associated with clinical side effects, 
      biochemical aberrations, significant changes in liver and kidney function, or 
      changes in serum levels of the hormones regulating mineral metabolism. In all 
      patients, a band-like metaphyseal sclerosis appeared on radiography of the long 
      bone. However, APD did not interfere with bone growth. CONCLUSIONS: The marked 
      clinical improvement in the clinical skeletal manifestations of Gaucher's disease 
      and the absence of toxic side effects in adolescent patients treated with APD 
      support previous findings in three adult patients on the efficacy of APD and 
      indicate possibilities for its use in inducing prolonged remissions in affected 
      patients.
FAU - Samuel, R
AU  - Samuel R
AD  - Department of Orthopedics, Beilinson Medical Center, Petah Tiqva, Israel.
FAU - Katz, K
AU  - Katz K
FAU - Papapoulos, S E
AU  - Papapoulos SE
FAU - Yosipovitch, Z
AU  - Yosipovitch Z
FAU - Zaizov, R
AU  - Zaizov R
FAU - Liberman, U A
AU  - Liberman UA
LA  - eng
PT  - Journal Article
PL  - United States
TA  - Pediatrics
JT  - Pediatrics
JID - 0376422
RN  - 0 (Diphosphonates)
RN  - OYY3447OMC (Pamidronate)
SB  - IM
MH  - Adolescent
MH  - Bone Diseases/*drug therapy/epidemiology/etiology
MH  - Diphosphonates/*therapeutic use
MH  - Female
MH  - Femur/diagnostic imaging
MH  - Follow-Up Studies
MH  - Fractures, Spontaneous/prevention & control
MH  - Gaucher Disease/complications/*drug therapy/epidemiology
MH  - Hand/diagnostic imaging
MH  - Humans
MH  - Male
MH  - Pamidronate
MH  - Radiography
MH  - Tibia/diagnostic imaging
MH  - Time Factors
EDAT- 1994/09/01 00:00
MHDA- 1994/09/01 00:01
CRDT- 1994/09/01 00:00
PHST- 1994/09/01 00:00 [pubmed]
PHST- 1994/09/01 00:01 [medline]
PHST- 1994/09/01 00:00 [entrez]
PST - ppublish
SO  - Pediatrics. 1994 Sep;94(3):385-9.