Osteoporoz tanılı çocuk hastalarda pamidronat tedavisinin etkinliği ve güvenilirliği
Küçük Resim Yok
Tarih
2020
Yazarlar
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
İnönü Üniversitesi
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
Amaç: Çocukluk çağında osteoporoz nadir görülse de iskelet deformiteleri ve hareket kabiliyetinde azalmaya neden olabilen önemli bir hastalıktır. Günümüzde osteoporoz tanılı çocukların tedavisinde en sık kullanılan ajan pamidronat olmasına rağmen halen FDA onayı yoktur. Çalışmamızda kliniğimizde izlenen osteoporoz tanılı çocuk hastalarda parenteral pamidronat tedavisinin etkinliğinin ve güvenilirliğinin araştırılması amaçlanmıştır. Gereç ve Yöntemler: Retrospektif çalışmamıza 2000-2019 yılları arasında İnönü Üniversitesi Tıp Fakültesi Çocuk Endokrinoloji Bilim Dalı'nda osteoporoz tanısı ile izlenen, 3 ay aralarla en az 6 ay parenteral pamidronat tedavisi alan, yeterli veri kayıtları olan 0-18 yaş arasındaki 25 hasta dahil edilmiştir. Hastaların tedavi öncesi ve tedavi altındaki 6. ay, 1. yıl, 2. yıl, 3. yıldaki antropometrik ölçümleri, laboratuvar verileri (serum Ca, P, ALP, 25OHD, PTH), yıllık kırık sayıları, aktivite skorları ve KMD Z-skorları karşılaştırıldı. Tedavi boyunca görülen yan etkiler değerlendirildi. Tüm veriler hastanın tıbbi kayıtlarından alındı. Bulgular: Osteoporoz tanılı hastaların ortalama tanı yaşları 7,69±5,35 yıl idi. Hastaların %60'ı (15/25) primer osteoporoz, diğerleri sekonder osteoporoz tanılıydı. Primer osteoporozlu hastalarda ortalama tanı yaşı daha düşük, kırık sayısı ve deformite sıklığı ise daha yüksek bulundu. Osteoporoz tanılı tüm hastaların pamidronat tedavisi öncesi ile tedavi sürecindeki VA SDS, boy SDS, VKİ SDS ve aktivite skorları arasında anlamlı fark saptanmadı. Hastalarda, tedavinin 6. ayında tedavi öncesine göre kırık sayısında anlamlı azalma saptanırken izleyen yıllarda bu farkın ortadan kalktığı görüldü. Tedavi süresince laboratuvar verileri normal sınırlar içinde seyretti. Hastaların tedavi sürecindeki 6.ay, 1.yıl, 2.yıl, ve 3.yıl ortalama KMD Z–skoru değerleri tedavi öncesine göre anlamlı olarak yüksek saptandı, yıllar içinde de anlamlı artış saptandı. Hastaların %32'sinde yan etki gözlendi, en sık yan etki asemptomatik hipokalsemiydi. Ciddi veya hayatı tehdit edici yan etki gözlenmedi. Sonuç: Çocukluk çağı osteoporozunda parenteral pamidronat tedavisi KMD Z-skorunu artırmada etkili ve güvenilir bir seçenektir. Anahtar Kelimeler: Çocuklarda osteoporoz, primer osteoporoz, sekonder osteoporoz, osteogenezis imperfekta, pamidronat, KMD Z- skoru.
Effectiveness and Safety of Pamidronate Treatment in Pediatric Patients with Osteoporosis Objective: Although it is rare in childhood, osteoporosis is an important disease that can cause skeletal deformities and reduced mobility. Although pamidronate is the most commonly used agent in the treatment of children diagnosed with osteoporosis today, it still does not have FDA approval. In our study, we aimed to investigate the effectiveness and safety of parenteral pamidronate treatment in pediatric patients with osteoporosis followed up in our clinic. Materials and Methods: Twenty-five patients between the ages of 0-18, who were followed up with the diagnosis of osteoporosis in the Pediatric Endocrinology Department of İnönü University Faculty of Medicine between 2000-2019, received at least 6 months of parenteral pamidronate treatment with 3-months intervals, and had sufficient data in medical records were included in our retrospective study. Anthropometric measurements, laboratory findings (serum Ca, P, ALP, 25OHD, PTH), annual number of fractures, activity scores and BMD Z-scores of the patients before treatment and at 6th month, 1st year, 2nd year and 3rd year under treatment were compared. Side effects seen during the treatment were evaluated. All data was obtained from the medical records of the patients. Results: The mean age at diagnosis of patients with osteoporosis was 7.69 ± 5.35 years. Sixty percent (15/25) of the patients were diagnosed with primary osteoporosis, and the others were diagnosed with secondary osteoporosis. Patients with primary osteoporosis were found to have a lower mean age, a higher number of fractures and also higher frequency of deformity at diagnosis. There was no significant difference between the BW SDS, height SDS, BMI SDS and activity scores of the all patients with osteoporosis before pamidronate treatment and during the treatment process. While there was a significant decrease in the number of fractures at the 6th month of the treatment compared to the pre-treatment, it was observed that this difference disappeared in the following years. Laboratory data remained within normal limits during the treatment period. Mean BMD Z-score values of the patients at 6th month, 1st, 2nd and 3rd year during the treatment process were found to be significantly higher than pre-treatment values, and also a significant increase was found over the years. Side effects were observed in 32% of the patients, the most common side effect was asymptomatic hypocalcemia. No serious or life-threatening side effects were observed. Conclusion: Parenteral pamidronate therapy is an effective and safe option to increase the BMD Z-score in childhood osteoporosis. Keywords: Childhood osteoporosis, primary osteoporosis, secondary osteoporosis, osteogenesis imperfecta, pamidronate, BMD Z-score.
Effectiveness and Safety of Pamidronate Treatment in Pediatric Patients with Osteoporosis Objective: Although it is rare in childhood, osteoporosis is an important disease that can cause skeletal deformities and reduced mobility. Although pamidronate is the most commonly used agent in the treatment of children diagnosed with osteoporosis today, it still does not have FDA approval. In our study, we aimed to investigate the effectiveness and safety of parenteral pamidronate treatment in pediatric patients with osteoporosis followed up in our clinic. Materials and Methods: Twenty-five patients between the ages of 0-18, who were followed up with the diagnosis of osteoporosis in the Pediatric Endocrinology Department of İnönü University Faculty of Medicine between 2000-2019, received at least 6 months of parenteral pamidronate treatment with 3-months intervals, and had sufficient data in medical records were included in our retrospective study. Anthropometric measurements, laboratory findings (serum Ca, P, ALP, 25OHD, PTH), annual number of fractures, activity scores and BMD Z-scores of the patients before treatment and at 6th month, 1st year, 2nd year and 3rd year under treatment were compared. Side effects seen during the treatment were evaluated. All data was obtained from the medical records of the patients. Results: The mean age at diagnosis of patients with osteoporosis was 7.69 ± 5.35 years. Sixty percent (15/25) of the patients were diagnosed with primary osteoporosis, and the others were diagnosed with secondary osteoporosis. Patients with primary osteoporosis were found to have a lower mean age, a higher number of fractures and also higher frequency of deformity at diagnosis. There was no significant difference between the BW SDS, height SDS, BMI SDS and activity scores of the all patients with osteoporosis before pamidronate treatment and during the treatment process. While there was a significant decrease in the number of fractures at the 6th month of the treatment compared to the pre-treatment, it was observed that this difference disappeared in the following years. Laboratory data remained within normal limits during the treatment period. Mean BMD Z-score values of the patients at 6th month, 1st, 2nd and 3rd year during the treatment process were found to be significantly higher than pre-treatment values, and also a significant increase was found over the years. Side effects were observed in 32% of the patients, the most common side effect was asymptomatic hypocalcemia. No serious or life-threatening side effects were observed. Conclusion: Parenteral pamidronate therapy is an effective and safe option to increase the BMD Z-score in childhood osteoporosis. Keywords: Childhood osteoporosis, primary osteoporosis, secondary osteoporosis, osteogenesis imperfecta, pamidronate, BMD Z-score.
Açıklama
Anahtar Kelimeler
Çocuk Sağlığı ve Hastalıkları, Child Health and Diseases
