Clinical burden and healthcare resource utilization associated with managing transfusion-dependent β-thalassemia in France

Figures & data

Figure 1. Patient attrition. Abbreviations: HPFH, hereditary persistence of fetal hemoglobin; HSCT, hematopoietic stem cell transplant; LTD, long-term disease; RBCT, red blood cell transfusion; SCD, sickle cell disease; SNDS, système national des données de santé; TDT, transfusion-dependent β-thalassemia. Note: Values presented in parentheses represent the proportion of patients with ≥1 inpatient claim or LTD database registration during the study period.

Table 1. Baseline demographics of patients with TDT.

	Patients with TDT (N = 331)
Mean age (years) at index date (SD)	26.1 (18.0)
Age categories (years) at index date, n (%)
0‒11	88 (26.6)
12‒17	38 (11.5)
18‒35	108 (32.6)
≥36	97 (29.3)
Sex, n (%)
Female	164 (49.5)
Male	167 (50.5)
Residence, n (%)
Metropolitan France	314 (94.9)
Overseas France	13 (3.9)
Unknown^a	–
LTD registration for β-thalassemia, n (%)	289 (87.3)
Mean follow-up time in years (SD; range)	4.9 (1.9; 1.0–6.9)

Abbreviations: LTD, long-term disease; SD, standard deviation; TDT, transfusion-dependent β-thalassemia.

^aPatient numbers <10 were masked (i.e. “-”) to protect patient confidentiality.

Table 2. Clinical complications in the follow-up period in patients with TDT.

Complication, n (%)	Patients with TDT (N = 331)
Cardiopulmonary complications^a	62 (18.7)
Arrythmia	38 (11.5)
Atrial fibrillation	34 (10.3)
Heart failure	27 (8.2)
Pulmonary hypertension	11 (3.3)
Endocrine complications	86 (26.0)
Diabetes	35 (10.6)
Hypogonadotropic hypogonadism	30 (9.1)
Hypoparathyroidism	25 (7.6)
Infertility	14 (4.2)
Osteoporosis	22 (6.7)
Hepatobiliary complications^b	75 (22.7)
Hypercoagulable state^c	15 (4.5)
Infections^d	17 (5.1)
Malignancy^e	14 (4.2)
Mental health complications^f	17 (5.1)
Renal complications^g	31 (9.4)
Splenomegaly	29 (8.8)
Urinary tract complications	14 (4.2)

Abbreviations: TDT, transfusion-dependent β-thalassemia.

^aIncluding arrhythmia, atrial fibrillation, heart failure (acute decompensated health failure and congestive heart failure), pericarditis, and pulmonary hypertension.

^bIncluding cirrhosis, fibrosis, gallstones, hepatic insufficiency, hepatitis B, hepatitis C, hepatomegaly, liver cancer, and portal hypertension.

^cIncluding deep vein thrombosis, pulmonary embolism, and venous thrombosis.

^dIncluding bacteremia, septicemia, and pneumococcal sepsis.

^eIncluding solid malignancy, leukemia, lymphoma, and myeloma.

^fIncluding anxiety and depression.

^gIncluding chronic kidney disease, renal failure or end-stage renal disease, and renal stones.

Figure 2. Mortality by age during follow-up.

Table 3. Treatment use in the follow-up period in patients with TDT.

	Patients with TDT (N = 331)
Treatment	Mean rate PPPY (SD)	Prevalence, n (%)
Folic acid	2.5 (3.3)	204 (61.6)
RBC transfusion episodes	13.5 (5.2)	331 (100)
Iron chelation therapy	11.2 (5.3)	322 (97.3)
Pain medications	2.6 (5.3)	260 (78.6)
NSAIDs	1.5 (2.8)	234 (70.7)
Opioids	1.1 (3.2)	206 (62.2)
Penicillin	1.7 (2.6)	285 (86.1)

Abbreviations: NSAID, non-steroidal anti-inflammatory drug; PPPY, per patient per year; RBC, red blood cell; SD, standard deviation; TDT, transfusion-dependent β-thalassemia.

Table 4. Treatment use in the follow-up period by age group.

	Age group
Treatment, mean rate PPPY (SD)	<18 years (n = 126)	≥18 years (n = 205)
Folic acid	3.0 (3.8)	2.1 (3.0)
Iron chelation therapy	12.3 (5.2)	10.6 (5.4)
Pain medications	1.3 (3.2)	3.5 (6.1)
NSAIDs	0.8 (1.9)	2.0 (3.2)
Opioids	0.4 (1.5)	1.5 (3.8)
Penicillin	2.1 (2.7)	1.5 (2.5)
RBC transfusion episodes	12.6 (3.9)	14.1 (5.7)

Abbreviations: NSAID, non-steroidal anti-inflammatory drug; PPPY, per patient per year; RBCT, red blood cell; SD, standard deviation.

Figure 3. HCRU during follow-up in patients with TDT. Abbreviations: ER, emergency room; HCRU, healthcare resource utilization; PPPY, per patient per year; SD, standard deviation; TDT, transfusion-dependent β-thalassemia. ^aPatients had a mean rate of 13.8 (SD: 6.1) day cases (<1 day), 1.0 (SD: 2.7) overnight stays (≥1 day), and 26.0 (SD: 49.4) total days hospitalized PPPY. ^bThe mean rate of ER visits PPPY that were followed by an inpatient hospitalization was 0.2 (SD: 0.4).

Data availability statement

Data are available from The French National Healthcare Data System. Submit requests to the National Institute of Health Data (INDS) https://www.snds.gouv.fr/SNDS/Accueil.